PMID

ASP cm-sirna ID

ASP cm-sirna (As)

Modification

Modification position

Inhibition / IC50 (mut)

Inhibition / IC50 (wt)

Unit

Cell line

Experimental method

Target

19412185

ASP cm-sirna_0001

GCTGCTGCTGCTGCTGCTG

Uncharged Amide backbone, K:D-Lysine

1*19

0.34 ± 0.03

1.2 ± 0.3

µM

GM04281

Western blot

HTT

19412185

ASP cm-sirna_0002

GCTGCTGCTGCTGCTGCTG

Uncharged Amide backbone, K:D-Lysine

1*19

2.1 ± 0.5

> 16

µM

GM04281

Western blot

HTT

19412185

ASP cm-sirna_0003

GCTGCTGCTGCTGCTGCTG

Uncharged Amide backbone, K:D-Lysine, R:D-Arginine

1*19

0.33 ± 0.04

0.64 ± 0.08

µM

GM04281

Western blot

HTT

19412185

ASP cm-sirna_0004

GCTGCTGCTGCTGCTG

Uncharged Amide backbone, K:D-Lysine

1*16

0.39 ± 0.11

1.4 ± 0.3

µM

GM04281

Western blot

HTT

19412185

ASP cm-sirna_0005

GCTGCTGCTGCTG-

Uncharged Amide backbone, K: D-Lysine

1*13

0.47 ± 0.18

2.2 ± 0.4

µM

GM04281

Western blot

HTT

19412185

ASP cm-sirna_0006

GCTGCTGCTGGAAGGACTT

Uncharged Amide backbone, K: D-Lysine

1*19

0.018 ± 0.010

0.055 ± 0.023

µM

GM04281

Western blot

HTT

19412185

ASP cm-sirna_0007

GGCGGCTGTTGCTGCTGCT

Uncharged Amide backbone, K:D-Lysine

1*19

0.96 ± 0.13

4.8 ± 1.8

µM

GM04281

Western blot

HTT

19412185

ASP cm-sirna_0008

GCTGCTGCTGTTGCTGCTT

Uncharged Amide backbone, K:D-Lysine

1*19

0.018 ± 0.010

0.055 ± 0.023

µM

GM04281

Western blot

HTT

19412185

ASP cm-sirna_0009

ATAGGTCCCGCTGCTGCTG

Uncharged Amide backbone, K:D-Lysine

3,6,9,12,15,18

0.96 ± 0.13

4.8 ± 1.8

µM

GM04281

Western blot

HTT

19412185

ASP cm-sirna_0010

GCTGCTGCTGCTGCTGCTG

Uncharged Amide backbone, K:D-Lysine

1*19

0.58 ± 0.07

1.2 ± 0.4

µM

GM04869

Western blot

HTT

19412185

ASP cm-sirna_0011

GCTGCTGCTGCTGCTGCTG

Uncharged Amide backbone, K:D-Lysine

1*19

2.3 ± 0.3

> 8

µM

GM04869

Western blot

HTT

19412185

ASP cm-sirna_0012

GCTGCTGCTGCTGCTGCTG

LNA*DNA bases

3,6,9,12,15,18*1,2,4,5,7,8,10,11,13,14,16,17,19

0.017 ± 0.007

> 0.1

µM

GM04281

Western blot

HTT

19412185

ASP cm-sirna_0013

GCTGCTGCTGGAAGGACTT

LNA*DNA bases

3,6,9,12,15,18*1,2,4,5,7,8,10,11,13,14,16,17,19

0.018 ± 0.010

0.055 ± 0.023

µM

GM04281

Western blot

HTT

19412185

ASP cm-sirna_0014

GGCGGCTGTTGCTGCTGCT

LNA*DNA bases

3,6,9,12,15,18*1,2,4,5,7,8,10,11,13,14,16,17,19

0.086 ± 0.038

> 0.1

µM

GM04281

Western blot

HTT

19412185

ASP cm-sirna_0015

GCUGCUGCUGCUGCUGCUG

s:Phosphorothioate

2,4,6,8,10,12,14,16,18,20

0.012 ± 0.004

0.024 ± 0.009

µM

GM04281

Western blot

HTT

19412185

ASP cm-sirna_0016

GCUGCUGCUGGAAGGACUU

s:Phosphorothioate

2,4,6,8,10,12,14,16,18,20

0.018 ± 0.010

0.055 ± 0.023

µM

GM04281

Western blot

HTT

19412185

ASP cm-sirna_0017

GGCGGCUGUUGCUGCUGCU

s:Phosphorothioate

2,4,6,8,10,12,14,16,18,20

0.086 ± 0.038

> 0.1

µM

GM04281

Western blot

HTT

19412185

ASP cm-sirna_0018

GAGGAAGAGGAGGAGGCCGA

s:Phosphorothioate

2,4,6,8,10,12,14,16,18,20

0.023 ± 0.032

>0.2

µM

GM04281

Western blot

HTT

22939619

ASP cm-sirna_0019

TCUGCUGCUGCUGCUGCUGAA

s:Phosphorothioate*5'-VP:5-(E)-vinyl-Phosphonate*f:2'-Fluoro*2-O-Me:2-O-Methyl*2-MOE:2-Methyoxyethyl

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19 * 20,21

0.08 ±0.01

>1

µM

GM04719

Western blot

HTT

22939619

ASP cm-sirna_0020

TCUGCUGCAGCUGCUGCUGAA

s:Phosphorothioate*5'-VP:5-(E)-vinyl-Phosphonate*f:2-Fluoro*2-O-Me:2-O-Methyl*2-MOE:2-Methyoxyethyl

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19 * 20,21

0.03±0.003

>1

µM

GM04719

Western blot

HTT

22939619

ASP cm-sirna_0021

TCUGCUGCUACUGCUGCUGAA

s:Phosphorothioate*5'-VP:5-(E)-vinyl-Phosphonate*f:2-Fluoro*2-O-Me:2-O-Methyl*2-MOE:2-Methyoxyethyl

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19 * 20,21

2.23±0.002

>1

µM

GM04719

Western blot

HTT

22939619

ASP cm-sirna_0022

TCUGCUGCUGCUGCUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

0.05±0.001

7.18±0.015

µM

GM04719

Western blot

HTT

22939619

ASP cm-sirna_0023

TCUACUGCUGCUGCUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

0.08

6.11

µM

GM04719

Western blot

HTT

22939619

ASP cm-sirna_0024

TCUGAUGCUGCUGCUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

1.72±0.002

>1

µM

GM04719

Western blot

HTT

22939619

ASP cm-sirna_0025

TCUGCAGCUGCUGCUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

ND

ND

ND

GM04719

Western blot

HTT

22939619

ASP cm-sirna_0026

TCUGCUACUGCUGCUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

1.12±0.001

5.11

µM

GM04719

Western blot

HTT

22939619

ASP cm-sirna_0027

TCUGCUGAUGCUGCUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

0.04±0.001

0.29

µM

GM04719

Western blot

HTT

22939619

ASP cm-sirna_0028

TCUGCUGCAGCUGCUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

0.049±0.008

9.4

µM

GM04719

Western blot

HTT

22939619

ASP cm-sirna_0029

TCUGCUGCUACUGCUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

1.78±0.035

>1

µM

GM04719

Western blot

HTT

22939619

ASP cm-sirna_0030

TCUGCUGCUUCUGCUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

1.53±0.002

>1

µM

GM04719

Western blot

HTT

22939619

ASP cm-sirna_0031

TCUGCUGCUGAUGCUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

1.28±0.001

>1

µM

GM04719

Western blot

HTT

22939619

ASP cm-sirna_0032

TCUGCUGCUGCAGCUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

0.003

0.07

µM

GM04719

Western blot

HTT

22939619

ASP cm-sirna_0033

TCUGCUGCUGCUACUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

0.042

>1

µM

GM04719

Western blot

HTT

22939619

ASP cm-sirna_0034

TCUGCUGCUGCUGCUACUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

0.081

0.273

µM

GM04719

Western blot

HTT

22939619

ASP cm-sirna_0035

TCUGCUGCAACUGCUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

0.063±0.005

>1

µM

GM04719

Western blot

HTT

22939619

ASP cm-sirna_0036

TCUGCUGCAAAUGCUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

0.033±0.005

>1

µM

GM04719

Western blot

HTT

22939619

ASP cm-sirna_0037

TCUGCUGAAAAUGCUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

11.8±0.01

>1

µM

GM04719

Western blot

HTT

22939619

ASP cm-sirna_0038

TCUACUGCUACUGCUACUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

0.223

>1

µM

GM04719

Western blot

HTT

22939619

ASP cm-sirna_0039

TCAGCUGUUGCUACUGUUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

ND

ND

ND

GM04719

Western blot

HTT

22939619

ASP cm-sirna_0040

TGCUGCUGCUGCUGCUGCUAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

19.4

>100

µM

GM04719

Western blot

HTT

22939619

ASP cm-sirna_0041

TCUGCUGCUGCUGCUGCUGAA

s:Phosphorothioate*5'-VP:5-(E)-vinyl-Phosphonate*f:2-Fluoro*2-O-Me:2-O-Methyl*2-MOE:2-Methyoxyethyl

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19 * 20,21

0.08 ±0.01

>1

µM

GM04281

Western blot

HTT

22939619

ASP cm-sirna_0042

TCUGCUGCAGCUGCUGCUGAA

s:Phosphorothioate*5'-VP:5-(E)-vinyl-Phosphonate*f:2-Fluoro*2-O-Me:2-O-Methyl*2-MOE:2-Methyoxyethyl

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19 * 20,21

0.03±0.003

>1

µM

GM04281

Western blot

HTT

22939619

ASP cm-sirna_0043

TCUGCUGCUACUGCUGCUGAA

s:Phosphorothioate*5'-VP:5-(E)-vinyl-Phosphonate*f:2-Fluoro*2-O-Me:2-O-Methyl*2-MOE:2-Methyoxyethyl

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19 * 20,21

2.23±0.002

>1

µM

GM04281

Western blot

HTT

22939619

ASP cm-sirna_0044

TCUGCUGCUGCUGCUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

0.05±0.001

7.18±0.015

µM

GM04281

Western blot

HTT

22939619

ASP cm-sirna_0045

TCUACUGCUGCUGCUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

0.08

6.11

µM

GM04281

Western blot

HTT

22939619

ASP cm-sirna_0046

TCUGAUGCUGCUGCUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

1.72±0.002

>1

µM

GM04281

Western blot

HTT

22939619

ASP cm-sirna_0047

TCUGCAGCUGCUGCUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

ND

ND

ND

GM04281

Western blot

HTT

22939619

ASP cm-sirna_0048

TCUGCUACUGCUGCUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

1.12±0.001

5.11

µM

GM04281

Western blot

HTT

22939619

ASP cm-sirna_0049

TCUGCUGAUGCUGCUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

0.04±0.001

0.29

µM

GM04281

Western blot

HTT

22939619

ASP cm-sirna_0050

TCUGCUGCAGCUGCUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

0.049±0.008

9.4

µM

GM04281

Western blot

HTT

22939619

ASP cm-sirna_0051

TCUGCUGCUACUGCUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

1.78±0.035

>1

µM

GM04281

Western blot

HTT

22939619

ASP cm-sirna_0052

TCUGCUGCUUCUGCUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

1.53±0.002

>1

µM

GM04281

Western blot

HTT

22939619

ASP cm-sirna_0053

TCUGCUGCUGAUGCUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

1.28±0.001

>1

µM

GM04281

Western blot

HTT

22939619

ASP cm-sirna_0054

TCUGCUGCUGCAGCUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

0.003

0.07

µM

GM04281

Western blot

HTT

22939619

ASP cm-sirna_0055

TCUGCUGCUGCUACUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

0.042

>1

µM

GM04281

Western blot

HTT

22939619

ASP cm-sirna_0056

TCUGCUGCUGCUGCUACUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

0.081

0.273

µM

GM04281

Western blot

HTT

22939619

ASP cm-sirna_0057

TCUGCUGCAACUGCUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

0.063±0.005

>1

µM

GM04281

Western blot

HTT

22939619

ASP cm-sirna_0058

TCUGCUGCAAAUGCUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

0.033±0.005

>1

µM

GM04281

Western blot

HTT

22939619

ASP cm-sirna_0059

TCUGCUGAAAAUGCUGCUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

11.8±0.01

>1

µM

GM04281

Western blot

HTT

22939619

ASP cm-sirna_0060

TCUACUGCUACUGCUACUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

0.223

>1

µM

GM04281

Western blot

HTT

22939619

ASP cm-sirna_0061

TCAGCUGUUGCUACUGUUGAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

ND

ND

ND

GM04281

Western blot

HTT

22939619

ASP cm-sirna_0062

TGCUGCUGCUGCUGCUGCUAA

5-Phosphate*2-Methyoxyethyl*s:Phosphorothioate*2-Fluoro*2-O-Methyl

1 * 1 * 1,2,4,6,8,10,12,14,15,16,17,18,19,20 * 2,4,6,8,10,12,14,16,18 * 3,5,7,9,11,13,15,17,19

19.4

>100

µM

GM04281

Western blot

HTT

22336946

ASP cm-sirna_0063

GGAGCCCGACCACAGCUGG

5′-Amine(sense)

1

18

5

µM

239T

DLR Assay

COL7A1

22336946

ASP cm-sirna_0064

GAGCCCGACCACAGCUGGC

5′-Amine(sense)

1

42

35

µM

239T

DLR Assay

COL7A1

22336946

ASP cm-sirna_0065

AGCCCGACCACAGCUGGCA

5′-Amine(sense)

1

40

38

µM

239T

DLR Assay

COL7A1

22336946

ASP cm-sirna_0066

CCGACCACAGCGGGCACAC

5′-Amine(sense)

1

70

48

µM

239T

DLR Assay

COL7A1

22336946

ASP cm-sirna_0067

GACCACAGCUGGCACACCA

5′-Amine(sense)

1

0

35

µM

239T

DLR Assay

COL7A1

22336946

ASP cm-sirna_0068

CGACCACAGCUGGCACACC

5′-Amine(sense),5-Phosphate(As)

1,1

38

50

µM

239T

DLR Assay

COL7A1

22336946

ASP cm-sirna_0069

CCCGACCACAGCUGGCACA

5′-Amine(sense),5-Phosphate(As)

1,1

ND

ND

ND

239T

DLR Assay

COL7A1

22336946

ASP cm-sirna_0070

ACAGCUGGCACACCAGGGC

5′-Amine(sense),5-Phosphate(As)

1,1

ND

ND

ND

239T

DLR Assay

COL7A1

22336946

ASP cm-sirna_0071

CCUGGGAAUACCAGGCUUU

5′-Amine(sense)

1

0

21

µM

239T

DLR Assay

COL7A1

22336946

ASP cm-sirna_0072

GCCUGGGAAUACCAGGCUU

5′-Amine(sense)

1

54

40

µM

239T

DLR Assay

COL7A1

22336946

ASP cm-sirna_0073

CUGGGAGCCUGGGAAUACC

5′-Amine(sense)

1

40

45

µM

239T

DLR Assay

COL7A1

22336946

ASP cm-sirna_0074

GCCUGGGAGCCUGGGAAUA

5′-Amine(sense)

1

3

8

µM

239T

DLR Assay

COL7A1

22336946

ASP cm-sirna_0075

AACCCUGCCUGGGAGUCUGGG

5′-Amine(sense)

1

5

10

µM

239T

DLR Assay

COL7A1

22336946

ASP cm-sirna_0076

GGAGCCCGACCACAGCUGG

5′-Amine(sense)

1

18

5

µM

Dermal fibroblasts

DLR Assay

COL7A1

22336946

ASP cm-sirna_0077

GAGCCCGACCACAGCUGGC

5′-Amine(sense)

1

42

35

µM

Dermal fibroblasts

DLR Assay

COL7A1

22336946

ASP cm-sirna_0078

AGCCCGACCACAGCUGGCA

5′-Amine(sense)

1

40

38

µM

Dermal fibroblasts

DLR Assay

COL7A1

22336946

ASP cm-sirna_0079

CCGACCACAGCGGGCACAC

5′-Amine(sense)

1

70

48

µM

Dermal fibroblasts

DLR Assay

COL7A1

22336946

ASP cm-sirna_0080

GACCACAGCUGGCACACCA

5′-Amine(sense)

1

0

35

µM

Dermal fibroblasts

DLR Assay

COL7A1

22336946

ASP cm-sirna_0081

CGACCACAGCUGGCACACC

5′-Amine(sense),5-Phosphate(As)

1,1

38

50

µM

Dermal fibroblasts

DLR Assay

COL7A1

22336946

ASP cm-sirna_0082

CCCGACCACAGCUGGCACA

5′-Amine(sense),5-Phosphate(As)

1,1

ND

ND

ND

Dermal fibroblasts

DLR Assay

COL7A1

22336946

ASP cm-sirna_0083

ACAGCUGGCACACCAGGGC

5′-Amine(sense),5-Phosphate(As)

1,1

ND

ND

ND

Dermal fibroblasts

DLR Assay

COL7A1

22336946

ASP cm-sirna_0084

CCUGGGAAUACCAGGCUUU

5′-Amine(sense)

1

0

21

µM

Dermal fibroblasts

DLR Assay

COL7A1

22336946

ASP cm-sirna_0085

GCCUGGGAAUACCAGGCUU

5′-Amine(sense)

1

54

40

µM

Dermal fibroblasts

DLR Assay

COL7A1

22336946

ASP cm-sirna_0086

CUGGGAGCCUGGGAAUACC

5′-Amine(sense)

1

40

45

µM

Dermal fibroblasts

DLR Assay

COL7A1

22336946

ASP cm-sirna_0087

GCCUGGGAGCCUGGGAAUA

5′-Amine(sense)

1

3

8

µM

Dermal fibroblasts

DLR Assay

COL7A1

22336946

ASP cm-sirna_0088

AACCCUGCCUGGGAGUCUGGG

5′-Amine(sense)

1

5

10

µM

Dermal fibroblasts

DLR Assay

COL7A1

22336946

ASP cm-sirna_0089

GGAGCCCGACCACAGCUGG

5′-Amine(sense)

1

18

5

µM

Keratinocytes

DLR Assay

COL7A1

22336946

ASP cm-sirna_0090

GAGCCCGACCACAGCUGGC

5′-Amine(sense)

1

42

35

µM

Keratinocytes

DLR Assay

COL7A1

22336946

ASP cm-sirna_0091

AGCCCGACCACAGCUGGCA

5′-Amine(sense)

1

40

38

µM

Keratinocytes

DLR Assay

COL7A1

22336946

ASP cm-sirna_0092

CCGACCACAGCGGGCACAC

5′-Amine(sense)

1

70

48

µM

Keratinocytes

DLR Assay

COL7A1

22336946

ASP cm-sirna_0093

GACCACAGCUGGCACACCA

5′-Amine(sense)

1

0

35

µM

Keratinocytes

DLR Assay

COL7A1

22336946

ASP cm-sirna_0094

CGACCACAGCUGGCACACC

5′-Amine(sense),5-Phosphate(As)

1,1

38

50

µM

Keratinocytes

DLR Assay

COL7A1

22336946

ASP cm-sirna_0095

CCCGACCACAGCUGGCACA

5′-Amine(sense),5-Phosphate(As)

1,1

ND

ND

ND

Keratinocytes

DLR Assay

COL7A1

22336946

ASP cm-sirna_0096

ACAGCUGGCACACCAGGGC

5′-Amine(sense),5-Phosphate(As)

1,1

ND

ND

ND

Keratinocytes

DLR Assay

COL7A1

22336946

ASP cm-sirna_0097

CCUGGGAAUACCAGGCUUU

5′-Amine(sense)

1

0

21

µM

Keratinocytes

DLR Assay

COL7A1

22336946

ASP cm-sirna_0098

GCCUGGGAAUACCAGGCUU

5′-Amine(sense)

1

54

40

µM

Keratinocytes

DLR Assay

COL7A1

22336946

ASP cm-sirna_0099

CUGGGAGCCUGGGAAUACC

5′-Amine(sense)

1

40

45

µM

Keratinocytes

DLR Assay

COL7A1

22336946

ASP cm-sirna_0100

GCCUGGGAGCCUGGGAAUA

5′-Amine(sense)

1

3

8

µM

Keratinocytes

DLR Assay

COL7A1

22336946

ASP cm-sirna_0101

AACCCUGCCUGGGAGUCUGGG

5′-Amine(sense)

1

5

10

µM

Keratinocytes

DLR Assay

COL7A1

22939618

ASP cm-sirna_0102

UUGUCUCUGGUCCUUACUU

5'-MP:-5'-methylenephosphonate*2'-MOE

1*1

ND

>1

µM

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0103

UUGUCUCUGGUCCUUACUU

5'-MP:-5'-methylenephosphonate*2'-MOE*s:Phosphorothioate

1*1*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19

>1

>1

µM

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0104

UUGUCUCUGGUCCUUACUU

5'-MP:5'-methylenephosphonate*2'-MOE*s:Phosphorothioate*2'-fluororibose(2'-F)

1*1*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19

>1

ND

µM

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0105

UUGUCUCUGGUCCUUACUU

5'-MP:5'-methylenephosphonate*2'-MOE*s:Phosphorothioate

1*1*12,13,14,15,16,17,18

0.39±0.009

ND

µM

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0106

UUGUCUCUGGUCCUUACUU

5'-MP:5'-methylenephosphonate*2'-MOE*s:Phosphorothioate*2'-fluororibose(2'-F)

1*1*12,13,14,15,16,17,18*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19

0.19±0.006

ND

µM

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0107

UUGUCUCUGGUCCUUACUUAA

5'-MP:5'-methylenephosphonate*2'-MOE*s:Phosphorothioate*2'-fluororibose(2'-F)*2'-MOE

1*1*12,13,14,15,16,17,18*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17*18,19

0.05±0.002

ND

µM

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0108

UUGUCUCUGGUCCUUACUUAA

5'-MP:5'-methylenephosphonate*2'-MOE*s:Phosphorothioate*2'-fluororibose(2'-F)*2'-MOE*2'-OMe

1*1*1,12,13,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*20,21*1,3,5,7,9,11,13,15,17,19

0.03±0.001

ND

µM

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0109

UUGUCUCUGGUCCUUACUUAA

5'-MP:5'-methylenephosphonate*2'-MOE*s:Phosphorothioate*2'-fluororibose(2'-F)*2'-MOE*2'-OMe

1*1*1,2,4,6,8,10,12,14,15,16,17,18,19*2,4,6,8,10,12,14,16,18*1,20,21*3,5,7,9,11,13,15,17,19

0.02±0.004

ND

µM

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0110

UUGUCUCUGGUCCUUACUU

5′-OH

1

0.005±0.0001

>0.9

µM

MEF

qRT_PCR

PTEN

22939618

ASP cm-sirna_0111

UUGUCUCUGGUCCUUACUU

5′-OH*s:Phosphorothioate

1*12,13,14,15,16,17,18

0.004

>1

µM

MEF

qRT_PCR

PTEN

22939618

ASP cm-sirna_0112

UUGUCUCUGGUCCUUACUU

5′-OH*s:Phosphorothioate*2'-F

1*12,13,14,15,16,17,18*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19

0.007

>0.8

µM

MEF

qRT_PCR

PTEN

22939618

ASP cm-sirna_0113

UUGUCUCUGGUCCUUACUUAA

5′-OH*s:Phosphorothioate*2'-F*2'-MOE

1*14,15,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*21*

0.006±0.0001

>0.7

µM

MEF

qRT_PCR

PTEN

22939618

ASP cm-sirna_0114

UUGUCUCUGGUCCUUACUUAA

5′-OH*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*1,20,21*1,3,5,7,9,11,13,15,17,19

0.009±0.0002

>0.6

µM

MEF

qRT_PCR

PTEN

22939618

ASP cm-sirna_0115

UUGUCUCUGGUCCUUACUU

5′-Ome

1

0.27±0.009

>0.4

µM

MEF

qRT_PCR

PTEN

22939618

ASP cm-sirna_0116

UUGUCUCUGGUCCUUACUUAA

5′-Ome*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome

1*2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*1,20,21*1,3,5,7,9,11,13,15,17,19

0.5

>0.3

µM

MEF

qRT_PCR

PTEN

22939618

ASP cm-sirna_0117

UUGUCUCUGGUCCUUACUU

5'-F

1

0.006±0.0001

ND

µM

MEF

qRT_PCR

PTEN

22939618

ASP cm-sirna_0118

UUGUCUCUGGUCCUUACUUAA

5'-F*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*1,20,21*1,3,5,7,9,11,13,15,17,19

0.013±0.0004

ND

µM

MEF

qRT_PCR

PTEN

22939618

ASP cm-sirna_0119

UUGUCUCUGGUCCUUACUU

5'deoxy(5'-H)

1

0.007±0.0002

ND

µM

MEF

qRT_PCR

PTEN

22939618

ASP cm-sirna_0120

UUGUCUCUGGUCCUUACUUAA

5'deoxy(5'-H)*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*1,20,21*1,3,5,7,9,11,13,15,17,19

0.011±0.0004

ND

µM

MEF

qRT_PCR

PTEN

22939618

ASP cm-sirna_0121

UUGUCUCUGGUCCUUACUU

5′-OH

1

0.005±0.0001

>0.9

µM

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0122

UUGUCUCUGGUCCUUACUU

5′-OH*s:Phosphorothioate

1*12,13,14,15,16,17,18

0.004

>1

µM

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0123

UUGUCUCUGGUCCUUACUU

5′-OH*s:Phosphorothioate*2'-F

1*12,13,14,15,16,17,18*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19

0.007

>0.8

µM

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0124

UUGUCUCUGGUCCUUACUUAA

5′-OH*s:Phosphorothioate*2'-F*2'-MOE

1*14,15,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*21*

0.006±0.0001

>0.7

µM

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0125

TUGUCUCUGGUCCUUACUUAA

5′-OH*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*1,20,21*1,3,5,7,9,11,13,15,17,19

0.009±0.0002

>0.6

µM

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0126

UUGUCUCUGGUCCUUACUU

5′-Ome

1

0.27±0.009

>0.4

µM

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0127

TUGUCUCUGGUCCUUACUUAA

5′-Ome*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome

1*2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*1,20,21*1,3,5,7,9,11,13,15,17,19

0.5

>0.3

µM

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0128

UUGUCUCUGGUCCUUACUU

5'-F

1

0.006±0.0001

ND

µM

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0129

TUGUCUCUGGUCCUUACUUAA

5'-F*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*1,20,21*1,3,5,7,9,11,13,15,17,19

0.013±0.0004

ND

µM

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0130

UUGUCUCUGGUCCUUACUU

5'deoxy(5'-H)

1

0.007±0.0002

ND

µM

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0131

TUGUCUCUGGUCCUUACUUAA

5'deoxy(5'-H)*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*1,20,21*1,3,5,7,9,11,13,15,17,19

0.011±0.0004

ND

µM

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0132

TUGUCUCUGGUCCUUACUUAA

5'-MP:5'-5'-methylenephosphonate*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*20,21*1,3,5,7,9,11,13,15,17,19

0.003

ND

µM

Hepatocytes

qRT_PCR

PTEN

22939618

ASP cm-sirna_0133

TUGUCUCUGGUCCUUACUUAA

5'-VP:5'-E-vinylphosphonate*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*20,21*1,3,5,7,9,11,13,15,17,19

0.011

ND

µM

Hepatocytes

qRT_PCR

PTEN

22939618

ASP cm-sirna_0134

TGAACAUUGGAAUAGUUUCAA

5'-P:-5'-Phosphate*2'-MOE*s:Phosphorothioate*2'-fluororibose(2'-F)*2'-MOE*2'-OMe

1*1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*1,20,21*1,3,5,7,9,11,13,15,17,19

0.001

ND

µM

Hepatocytes

qRT_PCR

PTEN

22939618

ASP cm-sirna_0135

TGAACAUUGGAAUAGUUUCAA

5'-MP:5'-methylenephosphonate*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*20,21*1,3,5,7,9,11,13,15,17,19

0.013

ND

µM

Hepatocytes

qRT_PCR

PTEN

22939618

ASP cm-sirna_0136

TGAACAUUGGAAUAGUUUCAA

5'-VP:5'-E-vinylphosphonate*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*20,21*1,3,5,7,9,11,13,15,17,19

0.004

ND

µM

Hepatocytes

qRT_PCR

PTEN

22939618

ASP cm-sirna_0137

TUAUCUAUAAUGAUCAGGUAA

5'-P:5'-Phosphate*2'-MOE*s:Phosphorothioate*2'-fluororibose(2'-F)*2'-MOE*2'-OMe

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*1,20,21*1,3,5,7,9,11,13,15,17,19

0.001

ND

µM

Hepatocytes

qRT_PCR

PTEN

22939618

ASP cm-sirna_0138

TUAUCUAUAAUGAUCAGGUAA

5'-MP:5'-methylenephosphonate*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*20,21*1,3,5,7,9,11,13,15,17,19

0.007

ND

µM

Hepatocytes

qRT_PCR

PTEN

22939618

ASP cm-sirna_0139

TUAUCUAUAAUGAUCAGGUAA

5'-VP:5'-E-vinylphosphonate*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*20,21*1,3,5,7,9,11,13,15,17,19

0.001

ND

µM

Hepatocytes

qRT_PCR

PTEN

22939618

ASP cm-sirna_0140

TUAUCUAUAAUGAUCAGGUAA

5'-VP:5'-E-vinylphosphonate*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome*C10

1*2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*1,20,21*1,3,5,7,9,11,13,15,17,19*8

0.002

ND

µM

Hepatocytes

qRT_PCR

PTEN

22939618

ASP cm-sirna_0141

TUAUCUAUAAUGAUCAGGUAA

5'-VP:5'-E-vinylphosphonate*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome*C16

1*2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*1,20,21*1,3,5,7,9,11,13,15,17,19*8

0.01

ND

µM

Hepatocytes

qRT_PCR

PTEN

22939618

ASP cm-sirna_0142

TAUCAAUGUAUUGGUUAAAA

5'-VP:5'-E-vinylphosphonate*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*20,21*1,3,5,7,9,11,13,15,17,19

>1

ND

µM

Hepatocytes

qRT_PCR

PTEN

22939618

ASP cm-sirna_0143

TUAAGACUUGAGAUGAUCCAA

5'-VP:5'-E-vinylphosphonate*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*20,21*1,3,5,7,9,11,13,15,17,19

0.002

ND

µM

Hepatocytes

qRT_PCR

Factor VII

22939618

ASP cm-sirna_0144

TAUGAGAACAUACUUUCCCAA

5'-VP:5'-E-vinylphosphonate*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*20,21*1,3,5,7,9,11,13,15,17,19

0.002

ND

µM

Hepatocytes

qRT_PCR

ApoCIII

22939618

ASP cm-sirna_0145

TUGUCUCUGGUCCUUACUUAA

5'-MP:5'-5'-methylenephosphonate*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*20,21*1,3,5,7,9,11,13,15,17,19

20

ND

%

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0146

TUGUCUCUGGUCCUUACUUAA

5'-VP:5'-E-vinylphosphonate*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*20,21*1,3,5,7,9,11,13,15,17,19

25

ND

%

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0147

TGAACAUUGGAAUAGUUUCAA

5'-P:-5'-Phosphate*2'-MOE*s:Phosphorothioate*2'-fluororibose(2'-F)*2'-MOE*2'-OMe

1*1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*1,20,21*1,3,5,7,9,11,13,15,17,19

ND

ND

%

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0148

TGAACAUUGGAAUAGUUUCAA

5'-MP:5'-methylenephosphonate*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*20,21*1,3,5,7,9,11,13,15,17,19

18

ND

%

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0149

TGAACAUUGGAAUAGUUUCAA

5'-VP:5'-E-vinylphosphonate*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*20,21*1,3,5,7,9,11,13,15,17,19

58

ND

%

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0150

TUAUCUAUAAUGAUCAGGUAA

5'-P:5'-Phosphate*2'-MOE*s:Phosphorothioate*2'-fluororibose(2'-F)*2'-MOE*2'-OMe

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*1,20,21*1,3,5,7,9,11,13,15,17,19

5

ND

%

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0151

TUAUCUAUAAUGAUCAGGUAA

5'-MP:5'-methylenephosphonate*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*20,21*1,3,5,7,9,11,13,15,17,19

38

ND

%

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0152

TUAUCUAUAAUGAUCAGGUAA

5'-VP:5'-E-vinylphosphonate*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*20,21*1,3,5,7,9,11,13,15,17,19

55

ND

%

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0153

TUAUCUAUAAUGAUCAGGUAA

5'-VP:5'-E-vinylphosphonate*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome*C10

1*2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*1,20,21*1,3,5,7,9,11,13,15,17,19*8

50

ND

%

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0154

TUAUCUAUAAUGAUCAGGUAA

5'-VP:5'-E-vinylphosphonate*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome*C16

1*2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*1,20,21*1,3,5,7,9,11,13,15,17,19*8

78

ND

%

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0155

TAUCAAUGUAUUGGUUAAAA

5'-VP:5'-E-vinylphosphonate*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*20,21*1,3,5,7,9,11,13,15,17,19

10

ND

%

Transgenic mouse model

qRT_PCR

PTEN

22939618

ASP cm-sirna_0156

TUAAGACUUGAGAUGAUCCAA

5'-VP:5'-E-vinylphosphonate*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*20,21*1,3,5,7,9,11,13,15,17,19

45

ND

%

Transgenic mouse model

qRT_PCR

Factor VII

22939618

ASP cm-sirna_0157

TAUGAGAACAUACUUUCCCAA

5'-VP:5'-E-vinylphosphonate*s:Phosphorothioate*2'-F*2'-MOE*2'-Ome

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*2,4,6,8,10,12,14,16,18*20,21*1,3,5,7,9,11,13,15,17,19

70

ND

%

Transgenic mouse model

qRT_PCR

ApoCIII

21028906

ASP cm-sirna_0158

GCTGCTGCTGCTGCTGCTG

LNA

3,6,9,12,15,18

0.4±0.07

>1

µM

GM042081

Western blot

HTT

21028906

ASP cm-sirna_0159

GCTGCTGCTGCTGCTGCTG

LNA

1,4,7,10,13,16,19

0.3±0.011

>1

µM

GM042081

Western blot

HTT

21028906

ASP cm-sirna_0160

GCTGCTGCTGCTGCTGCTG

LNA

2,5,8,11,14,17

>1

>1

µM

GM042081

Western blot

HTT

21028906

ASP cm-sirna_0161

CTGCTGCTGCTGCTGCTGC

LNA

2,5,8,11,14,17

0.64±0.026

>1

µM

GM042081

Western blot

HTT

21028906

ASP cm-sirna_0162

CTGCTGCTGCTGCTGCTGC

LNA

3,6,9,12,15,18

>1

>1

µM

GM042081

Western blot

HTT

21028906

ASP cm-sirna_0163

TGCTGCTGCTGCTGCTGCT

LNA

1,4,7,10,13,16,19

0.27±0.09

>1

µM

GM042081

Western blot

HTT

21028906

ASP cm-sirna_0164

TGCTGCTGCTGCTGCTGCT

LNA

3,6,9,12,15,18

>1

>1

µM

GM042081

Western blot

HTT

21028906

ASP cm-sirna_0165

GCTGCTGCTGCTGCTGCTGCTG

LNA

3,6,9,12,15,18,21

0.37±0.011

>1

µM

GM042081

Western blot

HTT

21028906

ASP cm-sirna_0166

GCTGCTGCTGCTGCTG

LNA

3,6,9,12,15

>1

>1

µM

GM042081

Western blot

HTT

21028906

ASP cm-sirna_0167

GCTGCTGCTGCTG

LNA

3,6,9,12

>1

>1

µM

GM042081

Western blot

HTT

21028906

ASP cm-sirna_0168

GCTGCTGCTGCTGCTGCTG

LNA

1,2,3,4,16,17,18,19

0.69±0.028

>1

µM

GM042081

Western blot

HTT

21028906

ASP cm-sirna_0169

GCTGCTGCTGCTGCTGCTG

LNA

3,6,9,12,15,18

0.4±0.07

>1

µM

GM04717

Western blot

HTT

21028906

ASP cm-sirna_0170

GCTGCTGCTGCTGCTGCTG

LNA

1,4,7,10,13,16,19

0.3±0.011

>1

µM

GM04717

Western blot

HTT

21028906

ASP cm-sirna_0171

GCTGCTGCTGCTGCTGCTG

LNA

2,5,8,11,14,17

>1

>1

µM

GM04717

Western blot

HTT

21028906

ASP cm-sirna_0172

CTGCTGCTGCTGCTGCTGC

LNA

2,5,8,11,14,17

0.64±0.026

>1

µM

GM04717

Western blot

HTT

21028906

ASP cm-sirna_0173

CTGCTGCTGCTGCTGCTGC

LNA

3,6,9,12,15,18

>1

>1

µM

GM04717

Western blot

HTT

21028906

ASP cm-sirna_0174

TGCTGCTGCTGCTGCTGCT

LNA

1,4,7,10,13,16,19

0.27±0.09

>1

µM

GM04717

Western blot

HTT

21028906

ASP cm-sirna_0175

TGCTGCTGCTGCTGCTGCT

LNA

3,6,9,12,15,18

>1

>1

µM

GM04717

Western blot

HTT

21028906

ASP cm-sirna_0176

GCTGCTGCTGCTGCTGCTGCTG

LNA

3,6,9,12,15,18,21

0.37±0.011

>1

µM

GM04717

Western blot

HTT

21028906

ASP cm-sirna_0177

GCTGCTGCTGCTGCTG

LNA

3,6,9,12,15

>1

>1

µM

GM04717

Western blot

HTT

21028906

ASP cm-sirna_0178

GCTGCTGCTGCTG

LNA

3,6,9,12

>1

>1

µM

GM04717

Western blot

HTT

21028906

ASP cm-sirna_0179

GCTGCTGCTGCTGCTGCTG

LNA

1,2,3,4,16,17,18,19

0.69±0.028

>1

µM

GM04717

Western blot

HTT

21028906

ASP cm-sirna_0180

GCUGCUGCUGCUGCUGCUG

cEt:5'-cEtbridgednucleicacid

3,6,9,12,15,18

0.33±0.007

>1

µM

GM042081

Western blot

HTT

21028906

ASP cm-sirna_0181

GCTGCTGCTGCTGCTGCTG

carba-LNA

2,5,8,11,14,17

0.15±0.008

>1

µM

GM042081

Western blot

HTT

21028906

ASP cm-sirna_0182

GCTGCTGCTGCTGCTGCTG

ENA:ethylenenucleicacid

3,6,9,12,15,18

>1

>1

µM

GM042081

Western blot

HTT

21028906

ASP cm-sirna_0183

CTGCTGCTGCTGCTGCTGC

ENA:ethylenenucleicacidonends

1,2,3,4,16,17,18,19

0.27±0.007

0.49±0.011

µM

GM042081

Western blot

HTT

21028906

ASP cm-sirna_0184

GCTGCTGCTGCTGCTGCTG

2'-MOE

1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19

>1

>1

µM

GM042081

Western blot

HTT

21028906

ASP cm-sirna_0185

GCUGCUGCUGCUGCUGCUG

2'-MOE*cEt:5'-cEtbridgednucleicacid

1,2,4,5,7,8,10,11,13,14,16,17,19*3,6,9,12,15,18

>1

>1

µM

GM042081

Western blot

HTT

21028906

ASP cm-sirna_0186

GCTGCTGCTGCTGCTGCTG

2'F-ANA:2'-flouroarabinonucleicacid

3,6,9,12,15,18

µM

GM042081

Western blot

HTT

21028906

ASP cm-sirna_0187

GCUGCUGCUGCUGCUGCUG

2'F-RNA:2'-flouroribonucleicacid

3,6,9,12,15,18

>1

>1

µM

GM042081

Western blot

HTT

21028906

ASP cm-sirna_0188

GCUGCUGCUGCUGCUGCUG

2'F-RNA:2'-flouroribonucleicacid

2,4,5,6,14,15,17,18

>1

>1

µM

GM042081

Western blot

HTT

21028906

ASP cm-sirna_0189

GCUGCUGCUGCUGCUGCUG

2'F-RNA:2'-flouroribonucleicacid

1,2,3,4,5,6,15,16,17,18

>1

>1

µM

GM042081

Western blot

HTT

21028906

ASP cm-sirna_0190

GCUGCUGCUGCUGCUGCUG

2'F-RNA:2'-flouroribonucleicacid

1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19

>1

>1

µM

GM042081

Western blot

HTT

21028906

ASP cm-sirna_0191

GCTGCUGCTGCUGCTGCUG

2'F-ANA:2'-flouroarabinonucleicacid

4,5,6,10,11,12,16,17,18

>1

>1

µM

GM042081

Western blot

HTT

21028906

ASP cm-sirna_0192

GCTGCTGCTGCTGCTGCTG

2'F-ANA:2'-flouroarabinonucleicacid

1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19

>1

>1

µM

GM042081

Western blot

HTT

21028906

ASP cm-sirna_0193

GCTGCTGCTGCTGCTGCTG

LNA*s:Phosphorothioate

3,6,9,12,18*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19

0.14±0.006

0.87±0.025

µM

GM042081

Western blot

HTT

21028906

ASP cm-sirna_0194

GCUGCUGCUGCUGCUGCUG

cEt:5'-cEtbridgednucleicacid*s:Phosphorothioate

3,6,9,12,18*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19

0.16±0.005

0.64±0.020

µM

GM042081

Western blot

HTT

21028906

ASP cm-sirna_0195

GCTGCTGCTGCTGCTGCTG

2'-MOE*s:Phosphorothioate

1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19

0.60±0.002

>1

µM

GM042081

Western blot

HTT

21028906

ASP cm-sirna_0196

GCUGCUGCUGCUGCUGCUG

cEt:5'-cEtbridgednucleicacid*s:Phosphorothioate*2'-MOE

3,6,9,12,18*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19*1,2,4,5,7,8,10,11,13,14,16,17

>1

>1

µM

GM042081

Western blot

HTT

21028906

ASP cm-sirna_0197

GmCUGCUGCUGCUGCUGCTG

2'F-ANA:2'-flouroarabinonucleicacid*2'-MOE*s:Phosphorothioate

3,4,5,6,7,8,9,10,11,12,13,14,15,16*1,2,18,19*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19

0.34±0.009

>1

µM

GM042081

Western blot

HTT

21028906

ASP cm-sirna_0198

GCTGCTGCTGCTGCTGCTG

carba-LNA*s:Phosphorothioate

2,5,8,11,14,17*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19

0.23±0.007

>1

µM

GM042081

Western blot

HTT

17940007

ASP cm-sirna_0199

UUCAUCAGCUUUUCCAGGGUC--CCC UGGAAAAGCUGAUGACGG-Chol

cholesterol-conjugated(cc)sensesiRNA*s:Phosphorothioate

19(s)*1,18,19(As)

60

70

%

Transgenic mouse model

Western blot

HTT

11809726

ASP cm-sirna_0200

CUGGAUGAGGAACAGCAACCUU:GGUUGCUGUUCCUCAUCCAGGA

5'-P:-5'-Phosphate

1(As),1(s)

60

20

%

S2

Northern blot

AR

11809726

ASP cm-sirna_0201

GAGUGAAUACCACGACGAUUUC:AAUCGUCGUGGUAUUCACUCCA

5'-P:-5'-Phosphate

1(As),1(s)

45

20

%

S2

Northern blot

AR

11809726

ASP cm-sirna_0202

GCAAGCUGACCCUGAAGUUCAU:GAACUUCAGGGUCAGCUUGCCG

5'-P:-5'-Phosphate

1(As),1(s)

20

5

%

S2

Northern blot

AR

11809726

ASP cm-sirna_0203

GCUCAAGGAUGGAAGUGCAGUU:CUGCACUUCCAUCCUUGAGCUU

5'-P:-5'-Phosphate

1(As),1(s)

80

30

%

S2

Northern blot

AR

11809726

ASP cm-sirna_0204

CUGGAUGAGGAACAGCAACCUU:GGUUGCUGUUCCUCAUCCAGGA

5'-P:-5'-Phosphate

1(As),1(s)

70

10

%

HEK293

Northern blot

AR

11809726

ASP cm-sirna_0205

GAGUGAAUACCACGACGAUUUC:AAUCGUCGUGGUAUUCACUCCA

5'-P:-5'-Phosphate

1(As),1(s)

50

20

%

HEK293

Northern blot

AR

11809726

ASP cm-sirna_0206

GCAAGCUGACCCUGAAGUUCAU:GAACUUCAGGGUCAGCUUGCCG

5'-P:-5'-Phosphate

1(As),1(s)

23

5

%

HEK293

Northern blot

AR

11809726

ASP cm-sirna_0207

GCUCAAGGAUGGAAGUGCAGUU:CUGCACUUCCAUCCUUGAGCUU

5'-P:-5'-Phosphate

1(As),1(s)

85

30

%

HEK293

Northern blot

AR

22859208

ASP cm-sirna_0208

GCGGTCAGCGATCCCAGGGT

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

44

20

%

HepG2

RT-PCR

hACTA1

22859208

ASP cm-sirna_0209

GGGAAGCGAGGCTTCACTTG

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

60

28

%

HepG2

RT-PCR

hACTA1

22859208

ASP cm-sirna_0210

CAGGGCTTTGTTTCGAAAAA

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

43

ND

%

HepG2

RT-PCR

hACTA1

22859208

ASP cm-sirna_0211

CCATTTTCTTCCACAGGGCT

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

28

10

%

HepG2

RT-PCR

hACTA1

22859208

ASP cm-sirna_0212

CAGAATGACTTTAATGCTTC

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

18

93

%

HepG2

RT-PCR

hACTA1

22859208

ASP cm-sirna_0213

GGGTCAGCTGCCAATGCTAG

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

60

40

%

HepG2

RT-PCR

Malat1

22859208

ASP cm-sirna_0214

CGGTGCAAGGCTTAGGAATT

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

ND

ND

ND

HepG2

RT-PCR

Malat1

22859208

ASP cm-sirna_0215

CTGCTAGCCTCTGGATTTGA

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

ND

96

%

HepG2

RT-PCR

Pten

22859208

ASP cm-sirna_0216

GCTTCAGTCATGACTTCCTT

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

80

82

%

HepG2

RT-PCR

Srb1

22859208

ASP cm-sirna_0217

CCTTCCCTGAAGGTTCCTCC

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

ND

20

%

HepG2

RT-PCR

NA

22859208

ASP cm-sirna_0218

TAGTGCGGACCTACCCACGA

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

10

5

%

HepG2

RT-PCR

NA

22859208

ASP cm-sirna_0219

CGGAGCGGTTGTGAACTGGC

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

56

34

%

HepG2

RT-PCR

hDMPK

22859208

ASP cm-sirna_0220

GCGGTCAGCGATCCCAGGGT

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

30

20

%

HepG2

Flourescence microscopy

hACTA1

22859208

ASP cm-sirna_0221

GGGAAGCGAGGCTTCACTTG

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

67

21

%

HepG2

Flourescence microscopy

hACTA1

22859208

ASP cm-sirna_0222

CAGGGCTTTGTTTCGAAAAA

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

50

10

%

HepG2

Flourescence microscopy

hACTA1

22859208

ASP cm-sirna_0223

CCATTTTCTTCCACAGGGCT

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

20

0

%

HepG2

Flourescence microscopy

hACTA1

22859208

ASP cm-sirna_0224

CAGAATGACTTTAATGCTTC

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

ND

93

%

HepG2

Flourescence microscopy

hACTA1

22859208

ASP cm-sirna_0225

GGGTCAGCTGCCAATGCTAG

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

44

12

%

HepG2

Flourescence microscopy

Malat1

22859208

ASP cm-sirna_0226

CGGTGCAAGGCTTAGGAATT

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

ND

ND

%

HepG2

Flourescence microscopy

Malat1

22859208

ASP cm-sirna_0227

CTGCTAGCCTCTGGATTTGA

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

ND

96

%

HepG2

Flourescence microscopy

Pten

22859208

ASP cm-sirna_0228

GCTTCAGTCATGACTTCCTT

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

80

82

%

HepG2

Flourescence microscopy

Srb1

22859208

ASP cm-sirna_0229

CCTTCCCTGAAGGTTCCTCC

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

ND

20

%

HepG2

Flourescence microscopy

NA

22859208

ASP cm-sirna_0230

TAGTGCGGACCTACCCACGA

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

ND

ND

%

HepG2

Flourescence microscopy

NA

22859208

ASP cm-sirna_0231

CGGAGCGGTTGTGAACTGGC

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

ND

ND

%

HepG2

Flourescence microscopy

hDMPK

22859208

ASP cm-sirna_0232

GCGGTCAGCGATCCCAGGGT

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

45

10

%

HAS-LR mice

Flourescence microscopy

hACTA1

22859208

ASP cm-sirna_0233

GGGAAGCGAGGCTTCACTTG

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

46

20

%

HAS-LR mice

Flourescence microscopy

hACTA1

22859208

ASP cm-sirna_0234

CAGGGCTTTGTTTCGAAAAA

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

33

10

%

HAS-LR mice

Flourescence microscopy

hACTA1

22859208

ASP cm-sirna_0235

CCATTTTCTTCCACAGGGCT

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

40

18

%

HAS-LR mice

Flourescence microscopy

hACTA1

22859208

ASP cm-sirna_0236

CAGAATGACTTTAATGCTTC

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

20

89

%

HAS-LR mice

Flourescence microscopy

hACTA1

22859208

ASP cm-sirna_0237

GGGTCAGCTGCCAATGCTAG

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

56

20

%

HAS-LR mice

Flourescence microscopy

Malat1

22859208

ASP cm-sirna_0238

CGGTGCAAGGCTTAGGAATT

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

ND

ND

ND

HAS-LR mice

Flourescence microscopy

Malat1

22859208

ASP cm-sirna_0239

CTGCTAGCCTCTGGATTTGA

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

ND

96

%

HAS-LR mice

Flourescence microscopy

Pten

22859208

ASP cm-sirna_0240

GCTTCAGTCATGACTTCCTT

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

80

82

%

HAS-LR mice

Flourescence microscopy

Srb1

22859208

ASP cm-sirna_0241

CCTTCCCTGAAGGTTCCTCC

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

ND

20

%

HAS-LR mice

Flourescence microscopy

NA

22859208

ASP cm-sirna_0242

TAGTGCGGACCTACCCACGA

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

ND

ND

ND

HAS-LR mice

Flourescence microscopy

NA

22859208

ASP cm-sirna_0243

CGGAGCGGTTGTGAACTGGC

2'-MOE*s:Phosphorothioate*5'deoxy(5'-H)

1,2,3,4,5,16,17,18,19,20*1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17,18,19,20*6,7,8,9,10,11,12,13,14,15,16

ND

ND

ND

HAS-LR mice

Flourescence microscopy

hDMPK

10666454

ASP cm-sirna_0244

CGACGCGATACCGCTTCTGC

ODNs:Antisenseoligodeoxynucleotides

11

5

ND

µM

MiaPaca II

Northern blot

POLR2A

10666454

ASP cm-sirna_0245

CGATACCGCTTCTGCCGGAC

ODNs:Antisenseoligodeoxynucleotides

5

<8

ND

µM

MiaPaca II

Northern blot

POLR2A

10666454

ASP cm-sirna_0246

CACGTCGACGCGATACCGCT

ODNs:Antisenseoligodeoxynucleotides

15

ND

ND

µM

MiaPaca II

Northern blot

POLR2A

10666454

ASP cm-sirna_0247

CGTCGACGCGATACCGCT

ODNs:Antisenseoligodeoxynucleotides

15

ND

ND

µM

MiaPaca II

Northern blot

POLR2A

10666454

ASP cm-sirna_0248

GTCGACGCGATACCGCT

ODNs:Antisenseoligodeoxynucleotides

15

<2

ND

µM

MiaPaca II

Northern blot

POLR2A

10666454

ASP cm-sirna_0249

TCGACGCGATACCGCT

ODNs:Antisenseoligodeoxynucleotides

15

3

ND

µM

MiaPaca II

Northern blot

POLR2A

10666454

ASP cm-sirna_0250

ACGTCGACGCGATACCGC

ODNs:Antisenseoligodeoxynucleotides

14

7

ND

µM

MiaPaca II

Northern blot

POLR2A

10666454

ASP cm-sirna_0251

CGTCGACGCGATACCGC

ODNs:Antisenseoligodeoxynucleotides

14

4

ND

µM

MiaPaca II

Northern blot

POLR2A

10666454

ASP cm-sirna_0252

GTCGACGCGATACCGC

ODNs:Antisenseoligodeoxynucleotides

14

>4

ND

µM

MiaPaca II

Northern blot

POLR2A

10666454

ASP cm-sirna_0253

TCGACGCGATACCGC

ODNs:Antisenseoligodeoxynucleotides

14

>1

ND

µM

MiaPaca II

Northern blot

POLR2A

10666454

ASP cm-sirna_0254

TCGACGCGATACCGCTT

ODNs:Antisenseoligodeoxynucleotides

11

>8

ND

µM

MiaPaca II

Northern blot

POLR2A

10666454

ASP cm-sirna_0255

CGACGCGATACCGCTT

ODNs:Antisenseoligodeoxynucleotides

11

>8

ND

µM

MiaPaca II

Northern blot

POLR2A

10666454

ASP cm-sirna_0256

CGACGCGATGCCGCTTCTGC

ODNs:Antisenseoligodeoxynucleotides

11

6

ND

µM

MiaPaca II

Northern blot

POLR2A

10666454

ASP cm-sirna_0257

CGATGCCGCTTCTGCCGGAC

ODNs:Antisenseoligodeoxynucleotides

5

<3

ND

µM

MiaPaca II

Northern blot

POLR2A

10666454

ASP cm-sirna_0258

CACGTCGACGCGATGCCGCT

ODNs:Antisenseoligodeoxynucleotides

15

<4

ND

µM

MiaPaca II

Northern blot

POLR2A

10666454

ASP cm-sirna_0259

CGTCGACGCGATGCCGCT

ODNs:Antisenseoligodeoxynucleotides

15

ND

ND

µM

MiaPaca II

Northern blot

POLR2A

10666454

ASP cm-sirna_0260

GTCGACGCGATGCCGCT

ODNs:Antisenseoligodeoxynucleotides

15

>8

ND

µM

MiaPaca II

Northern blot

POLR2A

10666454

ASP cm-sirna_0261

TCGACGCGATGCCGCT

ODNs:Antisenseoligodeoxynucleotides

15

>8

ND

µM

MiaPaca II

Northern blot

POLR2A

10666454

ASP cm-sirna_0262

ACGTCGACGCGATGCCGC

ODNs:Antisenseoligodeoxynucleotides

14

ND

ND

µM

MiaPaca II

Northern blot

POLR2A

10666454

ASP cm-sirna_0263

CGTCGACGCGATGCCGC

ODNs:Antisenseoligodeoxynucleotides

14

6

ND

µM

MiaPaca II

Northern blot

POLR2A

10666454

ASP cm-sirna_0264

GTCGACGCGATGCCGC

ODNs:Antisenseoligodeoxynucleotides

14

ND

ND

µM

MiaPaca II

Northern blot

POLR2A

10666454

ASP cm-sirna_0265

TCGACGCGATGCCGC

ODNs:Antisenseoligodeoxynucleotides

14

ND

ND

µM

MiaPaca II

Northern blot

POLR2A

10666454

ASP cm-sirna_0266

TCGACGCGATGCCGCTT

ODNs:Antisenseoligodeoxynucleotides

11

ND

ND

µM

MiaPaca II

Northern blot

POLR2A

10666454

ASP cm-sirna_0267

CGACGCGATGCCGCTT

ODNs:Antisenseoligodeoxynucleotides

11

>8

ND

µM

MiaPaca II

Northern blot

POLR2A

10666454

ASP cm-sirna_0268

CGACGCGATACCGCTTCTGC

ODNs:Antisenseoligodeoxynucleotides

11

5

ND

µM

15PC3

Northern blot

POLR2A

10666454

ASP cm-sirna_0269

CGATACCGCTTCTGCCGGAC

ODNs:Antisenseoligodeoxynucleotides

5

ND

ND

µM

15PC3

Northern blot

POLR2A

10666454

ASP cm-sirna_0270

CACGTCGACGCGATACCGCT

ODNs:Antisenseoligodeoxynucleotides

15

ND

ND

µM

15PC3

Northern blot

POLR2A

10666454

ASP cm-sirna_0271

CGTCGACGCGATACCGCT

ODNs:Antisenseoligodeoxynucleotides

15

ND

ND

µM

15PC3

Northern blot

POLR2A

10666454

ASP cm-sirna_0272

GTCGACGCGATACCGCT

ODNs:Antisenseoligodeoxynucleotides

15

<2

ND

µM

15PC3

Northern blot

POLR2A

10666454

ASP cm-sirna_0273

TCGACGCGATACCGCT

ODNs:Antisenseoligodeoxynucleotides

15

>8

ND

µM

15PC3

Northern blot

POLR2A

10666454

ASP cm-sirna_0274

ACGTCGACGCGATACCGC

ODNs:Antisenseoligodeoxynucleotides

14

ND

ND

µM

15PC3

Northern blot

POLR2A

10666454

ASP cm-sirna_0275

CGTCGACGCGATACCGC

ODNs:Antisenseoligodeoxynucleotides

14

>8

ND

µM

15PC3

Northern blot

POLR2A

10666454

ASP cm-sirna_0276

GTCGACGCGATACCGC

ODNs:Antisenseoligodeoxynucleotides

14

ND

ND

µM

15PC3

Northern blot

POLR2A

10666454

ASP cm-sirna_0277

TCGACGCGATACCGC

ODNs:Antisenseoligodeoxynucleotides

14

ND

ND

µM

15PC3

Northern blot

POLR2A

10666454

ASP cm-sirna_0278

TCGACGCGATACCGCTT

ODNs:Antisenseoligodeoxynucleotides

11

>8

ND

µM

15PC3

Northern blot

POLR2A

10666454

ASP cm-sirna_0279

CGACGCGATACCGCTT

ODNs:Antisenseoligodeoxynucleotides

11

>8

ND

µM

15PC3

Northern blot

POLR2A

10666454

ASP cm-sirna_0280

CGACGCGATGCCGCTTCTGC

ODNs:Antisenseoligodeoxynucleotides

11

ND

ND

µM

15PC3

Northern blot

POLR2A

10666454

ASP cm-sirna_0281

CGATGCCGCTTCTGCCGGAC

ODNs:Antisenseoligodeoxynucleotides

5

ND

ND

µM

15PC3

Northern blot

POLR2A

10666454

ASP cm-sirna_0282

CACGTCGACGCGATGCCGCT

ODNs:Antisenseoligodeoxynucleotides

15

ND

ND

µM

15PC3

Northern blot

POLR2A

10666454

ASP cm-sirna_0283

CGTCGACGCGATGCCGCT

ODNs:Antisenseoligodeoxynucleotides

15

ND

ND

µM

15PC3

Northern blot

POLR2A

10666454

ASP cm-sirna_0284

GTCGACGCGATGCCGCT

ODNs:Antisenseoligodeoxynucleotides

15

>8

ND

µM

15PC3

Northern blot

POLR2A

10666454

ASP cm-sirna_0285

TCGACGCGATGCCGCT

ODNs:Antisenseoligodeoxynucleotides

15

3

ND

µM

15PC3

Northern blot

POLR2A

10666454

ASP cm-sirna_0286

ACGTCGACGCGATGCCGC

ODNs:Antisenseoligodeoxynucleotides

14

ND

ND

µM

15PC3

Northern blot

POLR2A

10666454

ASP cm-sirna_0287

CGTCGACGCGATGCCGC

ODNs:Antisenseoligodeoxynucleotides

14

>8

ND

µM

15PC3

Northern blot

POLR2A

10666454

ASP cm-sirna_0288

GTCGACGCGATGCCGC

ODNs:Antisenseoligodeoxynucleotides

14

ND

ND

µM

15PC3

Northern blot

POLR2A

10666454

ASP cm-sirna_0289

TCGACGCGATGCCGC

ODNs:Antisenseoligodeoxynucleotides

14

ND

ND

µM

15PC3

Northern blot

POLR2A

10666454

ASP cm-sirna_0290

TCGACGCGATGCCGCTT

ODNs:Antisenseoligodeoxynucleotides

11

>8

ND

µM

15PC3

Northern blot

POLR2A

10666454

ASP cm-sirna_0291

CGACGCGATGCCGCTT

ODNs:Antisenseoligodeoxynucleotides

11

>8

ND

µM

15PC3

Northern blot

POLR2A

12527766

ASP cm-sirna_0292

UUUGUAGUGCCUGAAGCCG

s:Phosphorothioate

1,19

82

10

%

HaCaT

Northern blot

hTF

12527766

ASP cm-sirna_0293

UUUGUAGUGCCUGAAGCCG

s:Phosphorothioate

17,18,19

88

10

%

HaCaT

Northern blot

hTF

12527766

ASP cm-sirna_0294

UUUGUAGUGCCUGAAGCCG

s:Phosphorothioate

1,2,3,17,18,19

90

10

%

HaCaT

Northern blot

hTF

12527766

ASP cm-sirna_0295

UUUGUAGUGCCUGAAGCCG

s:Phosphorothioate

1,2,3,16,17,18,19

78

10

%

HaCaT

Northern blot

hTF

12527766

ASP cm-sirna_0296

UUUGUAGUGCCUGAAGCCG

2'-Ome

1,19

75

10

%

HaCaT

Northern blot

hTF

12527766

ASP cm-sirna_0297

UUUGUAGUGCCUGAAGCGC

2'-Ome

18,19

78

10

%

HaCaT

Northern blot

hTF

12527766

ASP cm-sirna_0298

UAUUUGUAGUGCCUGAAGC

2'-Ome

1,2,18,19

79

10

%

HaCaT

Northern blot

hTF

12527766

ASP cm-sirna_0299

UAUUUGUAGUGCCUGAAGC

2'-Ome

1,2,16,17,18,19

75

10

%

HaCaT

Northern blot

hTF

12527766

ASP cm-sirna_0300

UAUUUGUAGUGCCUGAAGC

2'-Allyl

1,19

80

10

%

HaCaT

Northern blot

hTF

12527766

ASP cm-sirna_0301

UUUGUAGUGCCUGAAGCGC

2'-Allyl

18,19

72

10

%

HaCaT

Northern blot

hTF

23887934

ASP cm-sirna_0302

CGACGACGACGACGACGAC

2'-Ome

1,4,7,10,13,16,19

ND

ND

ND

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0303

CGACGACGAUGACGACGAC

2'-Ome

1,4,7,10,13,16,19

ND

ND

ND

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0304

CGACGACGACUACGACGAC

2'-Ome

1,4,7,10,13,16,19

ND

ND

ND

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0305

UGCUGCUGCY34GCUGCUGCU:ACGACGACGACGACGACGA

2'-Ome*Abasicsite(2R,3S,4S)-2-hydroxy-methyl-4-methoxytetrahydrofuaran-3-ol(pentafuranoseabasicwith2-OMe,Y34)

2,5,7,10,13,16,18(sense),11(As)*10(As)

ND

ND

ND

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0306

UGCUGCUGCY33GCUGCUGCU:ACGACGACGACGACGACGA

2'-Ome*(2R,3S)-2-(hydroxymethyl)tetrahydro-2H-pyran-3-ol(hexapyranoseabasicmoiety,Y33)

2,5,7,10,13,16(sense),11(As)*10(As)

ND

ND

ND

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0307

GCUGCUGCY34GCUGCUGCUG:CGACGACGACGACGACGAC

2'-Ome*Abasicsite(2R,3S,4S)-2-hydroxy-methyl-4-methoxytetrahydrofuaran-3-ol(pentafuranoseabasicwith2-OMe,Y34)

1,4,7,10,12,14,17(sense),12(As)*11(As)

.003. ± 0.0004

0.075±0.03

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0308

GCUGCUGCUY34CUGCUGCUG:CGACGACGAUGACGACGAC

2'-Ome*Abasicsite(2R,3S,4S)-2-hydroxy-methyl-4-methoxytetrahydrofuaran-3-ol(pentafuranoseabasicwith2-OMe,Y34)

1,4,7,10,12,14,17(sense),12(As)*10(As)

.0037 ± .00013

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0309

GCUGCUGCUGY34UGCUGCUG:CGACGACGACUACGACGAC

2'-Ome*Abasicsite(2R,3S,4S)-2-hydroxy-methyl-4-methoxytetrahydrofuaran-3-ol(pentafuranoseabasicwith2-OMe,Y34)

1,4,7,10,12,14,17(sense),12(As)*9(As)

.0025 ± .0005

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0310

GCUGCUGCY33GCUGCUGCUG:CGACGACGACGACGACGAC

2'-Ome*(2R,3S)-2-(hydroxymethyl)tetrahydro-2H-pyran-3-ol(hexapyranoseabasicmoiety,Y33)

1,4,7,10,12,14,17(sense),12(As)*11(As)

.0028 ± .0004

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0311

GCUGCUGCUY33CUGCUGCUG:CGACGACGAUGACGACGAC

2'-Ome*(2R,3S)-2-(hydroxymethyl)tetrahydro-2H-pyran-3-ol(hexapyranoseabasicmoiety,Y33)

1,4,7,10,12,14,17(sense),12(As)*10(As)

.0058 ± .004

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0312

GCUGCUGCUGY33UGCUGCUG:CGACGACGACUACGACGAC

2'-Ome*(2R,3S)-2-(hydroxymethyl)tetrahydro-2H-pyran-3-ol(hexapyranoseabasicmoiety,Y33)

1,4,7,10,12,14,17(sense),12(As)*9(As)

.0033 ± .0006

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0313

GCUGCUGCY16Y16CUGCUGCUG:TCGACGACGAAGACGACGAC

2'-Ome*(2R,3S)-2-(hydroxymethyl)tetrahydrofuran-3-ol(pentafuranoseabasicwith2-deoxy,Y16)

12*11,12

.0061 ± .0014

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0314

GCUGCUGCY16Y16Y16UGCUGCUG:CGACGACGAAAACGACGAC

2'-Ome*(2R,3S)-2-(hydroxymethyl)tetrahydrofuran-3-ol(pentafuranoseabasicwith2-deoxy,Y16)

12*9,10,11

.029 ± .085

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0315

GCUGCUGCY16Y16Y16Y16GCUGCUG:CGACGACGAAAACGACGAC

2'-Ome*(2R,3S)-2-(hydroxymethyl)tetrahydrofuran-3-ol(pentafuranoseabasicwith2-deoxy,Y16)

12*8,9,10,11

.049 ± .065

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0316

GCUGCUGCY33Y33CUGCUGCUG:CGACGACGAAGACGACGAC

2'-Ome*(2R,3S)-2-(hydroxymethyl)tetrahydro-2H-pyran-3-ol(hexapyranoseabasicmoiety,Y33)

12*10,11

.0034 ± .001

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0317

GCUGCUGCY33Y33Y33UGCUGCUG:TCGACGACGAAAACGACGAC

2'-Ome*(2R,3S)-2-(hydroxymethyl)tetrahydro-2H-pyran-3-ol(hexapyranoseabasicmoiety,Y33)

12*10,11

.015 ± .042

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0318

GCUGCUGCY33Y33Y33Y33GCUGCUG:TCGACGACGAAAACGACGAC

2'-Ome*(2R,3S)-2-(hydroxymethyl)tetrahydro-2H-pyran-3-ol(hexapyranoseabasicmoiety,Y33)

12*8,9,10,11

.034 ± .011

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0319

GCUGCUGY33Y33GY33UGCUGCUG:CGACGACAACAACGACGAC

(2R,3S)-2-(hydroxymethyl)tetrahydro-2H-pyran-3-ol(hexapyranoseabasicmoiety,Y33)

9,11,12

.043 ± .074

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0320

GCUGCUGY33Y33Y33Y33UGCUGCUG:CGACGACAAAAACGACGAC

(2R,3S)-2-(hydroxymethyl)tetrahydro-2H-pyran-3-ol(hexapyranoseabasicmoiety,Y33)

9,10,11,12

>1

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0321

GCUGCUGCUGY33UGCUY33CUG:CGACGACGACAACGAAGAC

(2R,3S)-2-(hydroxymethyl)tetrahydro-2H-pyran-3-ol(hexapyranoseabasicmoiety,Y33)

4,9

.047 ± .015

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0322

UGCUGCUY33Y33UY33CUGCUGCUGCU:ACGACGAAAAAGACGACGACGA

2'-Ome*(2R,3S)-2-(hydroxymethyl)tetrahydro-2H-pyran-3-ol(hexapyranoseabasicmoiety,Y33)

13,16*12,14,15

.0052 ± .0022

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0323

UGCUGCUY33Y33Y33GCUGCUGCUGCU:ACGACGAAAACGACGACGACGA

2'-Ome*(2R,3S)-2-(hydroxymethyl)tetrahydro-2H-pyran-3-ol(hexapyranoseabasicmoiety,Y33)

16*13,14,15

.0038 ± .0014

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0324

UGCUGCUY16Y16UY16CUGCUGCUGCU:ACGACGAAAAAGACGACGACGA

2'-Ome*(2R,3S)-2-(hydroxymethyl)tetrahydrofuran-3-ol(pentafuranoseabasicwith2-deoxy,Y16)

13,16*12,14,15

.020 ± .0024

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0325

UGCUGCUY16Y16Y16GCUGCUGCUGCU:ACGACGAAAACGACGACGACGA

2'-Ome*(2R,3S)-2-(hydroxymethyl)tetrahydrofuran-3-ol(pentafuranoseabasicwith2-deoxy,Y16)

16*13,14,15

.011 ± .0015

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0326

GCUGCUGCAGCUGCUGCUG:CGACGACGUCGACGACGAC

2'-OMe

12(sense),1,4,7,10,13,16,19(As)

0.0028 ± 0.0002

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0327

GCUGCUGCAACUGCUGCUG:CGACGACGUUGACGACGAC

2'-OMe

12(sense),1,4,7,9,10,13,16,19(As)

0.0028 ± 0.0003

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0328

GCUGCUGCAGCUGCUGCUG:CGACGACGACGACGACGAC

2'-OMe

12(sense),1,4,7,10,13,16,19(As)

0.0033 ± 0.0003

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0329

GCUGCUGCUACUGCUGCUG:CGACGACGACGACGACGAC

2'-OMe

11(sense),1,4,7,10,13,16,19(As)

0.0068 ± 0.0007

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0330

GCUGCUGCUGAUGCUGCUG:CGACGACGACGACGACGAC

2'-OMe

1,4,7,10,13,16,19(As)

0.0049 ± 0.0006

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0331

CUGCUGCUGAUGCUGCUGC:GACGACGACGACGACGACG

2'-OMe

3,6,9,12,15,18(As)

0.0071± 0.0001

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0332

UGCUGCUGCAGCUGCUGCU:UGCUGCUGCAGCUGCUGCU

2'-OMe

11(sense),9(As)

0.0051± 0.0001

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0333

GCUGCUGAUACUGCUGCUG:CGACGACUAUGACGACGAC

2'-OMe

11(sense),4,7,8,10,16,19(As)

ND

ND

ND

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0334

GCUGCUGAUGAUGCUGCUG:CGACGACUACUACGACGAC

2'-OMe

4,7,8,10,11,16,19

ND

ND

ND

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0335

GCUGCUGAAACUGCUGCUG:CGACGACUUUGACGACGAC

2'-OMe

4,7,8,9,10,16,19

0.0051± 0.0006

.084±0.001

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0336

GCUGCUGAAGAUGCUGCUG:CGACGACUUCUACGACGAC

2'-OMe

4,7,8,9,10,11,16,19

0.0033± 0.0007

.067±0.0012

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0337

GCUGCUGCAAAUGCUGCUG:CGACGACGUUUACGACGAC

2'-OMe

12(sense),4,7,9,10,11,16,19(As)

0.0014± 0.0005

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0338

GCUGCUGAAAAUGCUGCUG:CGACGACUUUUACGACGAC

2'-OMe

4,7,8,10,11,16,19

0.0036± 0.00013

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0339

CUGCUGCAAAUGCUGCUGC:GACGACGUUUACGACGACG

2'-OMe

13(sense),3,6,8,9,10,12,15,18(As)

0.020± 0.0027

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0340

CUGCUGCAACAGCUGCUGC:GACGACGUUGUCGACGACG

2'-OMe

10(sense),3,6,8,9,10,11,12,15,18(As)

ND

ND

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0341

CUGCUGCUAAAGCUGCUGC:GACGACGAUUUCGACGACG

2'-OMe

12(sense),3,6,9,10,11,12,15,18(As)

ND

ND

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0342

CUGCUGCAAAAGCUGCUGC:GACGACGUUUUCGACGACG

2'-OMe

13(sense),3,6,8,9,10,11,12,15,18(As)

0.019± 0.0042

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0343

UGCUGCUAAUGCUGCUGCU:ACGACGAUUACGACGACGA

2'-OMe

13(sense),5,8,9,10,11,17(As)

ND

ND

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0344

UGCUGCUGAUACUGCUGCU:ACGACGACUAUGACGACGA

2'-OMe

10(sense),5,8,9,11,17(As)

ND

ND

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0345

UGCUGCUAAAGCUGCUGCU:ACGACGAUUUCGACGACGA

2'-OMe

13(sense),5,8,9,10,11,17(As)

0.0028± 0.005

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0346

UGCUGCUAAUACUGCUGCU:ACGACGAUUAUGACGACGA

2'-OMe

10,14(sense),5,8,9,11,17

0.0086± 0.0041

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0347

UGCUGCUGAAACUGCUGCU:UGCUGCUGAAACUGCUGCU

2'-OMe

1,3,6(As)

0.0027± 0.0011

>1

µM

GM04281

RT-PCR

HTT

23887934

ASP cm-sirna_0348

UGCUGCUAAAACUGCUGCU:ACGACGAUUUUGACGACGA

2'-OMe

13(sense),5,8,9,10,11,17(As)

0.0079± 0.0032

>1

µM

GM04281

RT-PCR

HTT

23935115

ASP cm-sirna_0349

TCUGCUGCAGCUGCUGCUGAA

5'VP*s:Phosphorothioate(PS-linkage)*2′-Ome*2'-F*,2'-MOE

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*3,5,79,11,13,15,17,19*2,4,6,810,12,14,16,18*1,20,21

0.0003± 0.0014

0.002± 0.0032

µM

Fibroblast

Western blot

ATXN3

23935115

ASP cm-sirna_0350

TCUGCUGCAGCUGCUGCUGAA

5'VP*Phosphorthioate(PS)linkage*2′-Ome*2'-F*,2'-MOE

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*3,5,79,11,13,15,17,19*2,4,6,810,12,14,16,18*1,20,21

0.0006± 0.0014

0.04± 0.0032

µM

GM04281

Western blot

ATXN3

23935115

ASP cm-sirna_0351

TCUGCUGCAGCUGCUGCUGAA

5'VP*Phosphorthioate(PS)linkage*2′-Ome*2'-F*,2'-MOE

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*3,5,79,11,13,15,17,19*2,4,6,810,12,14,16,18*1,20,21

0.0002± 0.0014

0.06± 0.0032

µM

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0352

TCUGCUGCAGCUGCUGCUGAA

5'VP*Phosphorthioate(PS)linkage*2′-Ome*2'-F*,2'-MOE

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*3,5,79,11,13,15,17,19*2,4,6,810,12,14,16,18*1,20,21

0.0002± 0.0014

0.08± 0.0032

µM

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0353

TCUACUGCUGCUGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

High efficacy

Low efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0354

TCUGAUGCUGCUGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

High efficacy

Low efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0355

TCUGCAGCUGCUGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

Moderate efficacy

Low efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0356

TCUGCUACUGCUGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

High efficacy

Low efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0357

TCUGCUGAUGCUGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

High efficacy

Low efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0358

TCUGCUGCAGCUGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

Highest efficacy

Lowest efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0359

TCUGCUGCUACUGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

Moderate efficacy

Lowest efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0360

TCUGCUGCUUCUGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

Low efficacy

Low efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0361

TCUGCUGCUGAUGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

Lowest efficacy

Low efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0362

TCUGCUGCUGCAGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

Moderate efficacy

Lowest efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0363

TCUGCUGCUGCUACUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

High efficacy

Low efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0364

TCUGCUGCUGCUGCUACUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

Highest efficacy

Lowest efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0365

TCUGCUGCAACUGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

High efficacy

Low efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0366

TCUGCUGCAAAUGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

High efficacy

Low efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0367

TCUGCUGAAAAUGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

Moderate efficacy

Lowest efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0368

TCUACUGCUACUGCUACUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

Low efficacy

Low efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0369

TCAGCUGUUGCUACUGUUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,17,19

Lowest efficacy

Lowest efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0370

TGCUGCUGCUGCUGCUGCUAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,17,19

Lowest efficacy

Lowest efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0371

TCUGCUGCAGCUGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

Highest efficacy

Lowest efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0372

TCUGCUGCAGCUGCUGCUAA

2'-MOE*2'-F*2'-Ome

1,19,20*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17

High efficacy

Low efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0373

TCUGCUGCAGCUGCUGCAA

2'-MOE*2'-F*2'-Ome

1,18,19*2,4,6,8,10,12,14,16*3,5,7,9,11,13,15,17

Moderate efficacy

Lowest efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0374

TCUGCUGCAGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,17,18*2,4,6,8,10,12,14,16*3,5,7,9,11,13,15

Moderate efficacy

Lowest efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0375

TCUGCUGCAGCUGCUAA

2'-MOE*2'-F*2'-Ome

1,16,17*2,4,6,8,10,12,14,*3,5,7,9,11,13,15

Low efficacy

Low efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0376

TCUGCUGCAGCUGCAA

2'-MOE*2'-F*2'-Ome

1,15,16*2,4,6,8,10,12,14,*3,5,7,9,11,13

Low efficacy

Low efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0377

TCUGCUGCAGCUGAA

2'-MOE*2'-F*2'-Ome

1,14,15*2,4,6,8,10,12,*3,5,7,9,11,13

Lowest efficacy

Lowest efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0378

TCUGCUGCAGAUGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,17,19

High efficacy

Low efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0379

TCUGCUGCAGCAGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,17,19

Moderate efficacy

Low efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0380

TCUGCUGCAGCUACUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,17,19

High efficacy

Low efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0381

TCUGCUGCAGCUGAUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,17,19

High efficacy

Low efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0382

TCUGCUGCAGCUGCAGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,17,19

Low efficacy

Low efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0383

TCUGCUGCAGCUGCUCCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,17,19

High efficacy

Low efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0384

TCUGCUGCAGCUGCUGAUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,17,19

High efficacy

Low efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0385

TCUGCUGCAGCUGCUGCAGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,17,19

Low efficacy

Low efficacy

%

GM06151

Western blot

ATXN3

23935115

ASP cm-sirna_0386

TCUGCUGCAGCUGCUGCUGAA

5'VP*s:Phosphorothioate*2'MOE*2'-F*,2′-Ome

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*3,5,79,11,13,15,17,19*2,4,6,810,12,14,16,18*1,20,21

0.0003± 0.0014

0.002± 0.0032

µM

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0387

TCUGCUGCAGCUGCUGCUGAA

5'VP*Phosphorthioate(PS)linkage*2'MOE*2'-F*,2′-Ome

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*3,5,79,11,13,15,17,19*2,4,6,810,12,14,16,18*1,20,21

0.0006± 0.0014

0.04± 0.0032

µM

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0388

TCUGCUGCAGCUGCUGCUGAA

5'VP*Phosphorthioate(PS)linkage*2'MOE*2'-F*,2′-Ome

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*3,5,79,11,13,15,17,19*2,4,6,810,12,14,16,18*1,20,21

0.0002± 0.0014

0.06± 0.0032

µM

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0389

TCUGCUGCAGCUGCUGCUGAA

5'VP*Phosphorthioate(PS)linkage*2'MOE*2'-F*,2′-Ome

1*1,2,4,6,8,10,12,14,15,16,17,18,19,20*3,5,79,11,13,15,17,19*2,4,6,810,12,14,16,18*1,20,21

0.0002± 0.0014

0.08± 0.0032

µM

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0390

TCUACUGCUGCUGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

High efficacy

Low efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0391

TCUGAUGCUGCUGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

High efficacy

Low efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0392

TCUGCAGCUGCUGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

Moderate efficacy

Low efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0393

TCUGCUACUGCUGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

High efficacy

Low efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0394

TCUGCUGAUGCUGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

High efficacy

Low efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0395

TCUGCUGCAGCUGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

Highest efficacy

Lowest efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0396

TCUGCUGCUACUGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

Moderate efficacy

Lowest efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0397

TCUGCUGCUUCUGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

Low efficacy

Low efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0398

TCUGCUGCUGAUGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

Lowest efficacy

Low efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0399

TCUGCUGCUGCAGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

Moderate efficacy

Lowest efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0400

TCUGCUGCUGCUACUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

High efficacy

Low efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0401

TCUGCUGCUGCUGCUACUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

Highest efficacy

Lowest efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0402

TCUGCUGCAACUGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

High efficacy

Low efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0403

TCUGCUGCAAAUGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

High efficacy

Low efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0404

TCUGCUGAAAAUGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

Moderate efficacy

Lowest efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0405

TCUACUGCUACUGCUACUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

Low efficacy

Low efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0406

TCAGCUGUUGCUACUGUUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,17,19

Lowest efficacy

Lowest efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0407

TGCUGCUGCUGCUGCUGCUAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,17,19

Lowest efficacy

Lowest efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0408

TCUGCUGCAGCUGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17,19

Highest efficacy

Lowest efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0409

TCUGCUGCAGCUGCUGCUAA

2'-MOE*2'-F*2'-Ome

1,19,20*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,15,17

High efficacy

Low efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0410

TCUGCUGCAGCUGCUGCAA

2'-MOE*2'-F*2'-Ome

1,18,19*2,4,6,8,10,12,14,16*3,5,7,9,11,13,15,17

Moderate efficacy

Lowest efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0411

TCUGCUGCAGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,17,18*2,4,6,8,10,12,14,16*3,5,7,9,11,13,15

Moderate efficacy

Lowest efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0412

TCUGCUGCAGCUGCUAA

2'-MOE*2'-F*2'-Ome

1,16,17*2,4,6,8,10,12,14,*3,5,7,9,11,13,15

Low efficacy

Low efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0413

TCUGCUGCAGCUGCAA

2'-MOE*2'-F*2'-Ome

1,15,16*2,4,6,8,10,12,14,*3,5,7,9,11,13

Low efficacy

Low efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0414

TCUGCUGCAGCUGAA

2'-MOE*2'-F*2'-Ome

1,14,15*2,4,6,8,10,12,*3,5,7,9,11,13

Lowest efficacy

Lowest efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0415

TCUGCUGCAGAUGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,17,19

High efficacy

Low efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0416

TCUGCUGCAGCAGCUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,17,19

Moderate efficacy

Low efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0417

TCUGCUGCAGCUACUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,17,19

High efficacy

Low efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0418

TCUGCUGCAGCUGAUGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,17,19

High efficacy

Low efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0419

TCUGCUGCAGCUGCAGCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,17,19

Low efficacy

Low efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0420

TCUGCUGCAGCUGCUCCUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,17,19

High efficacy

Low efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0421

TCUGCUGCAGCUGCUGAUGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,17,19

High efficacy

Low efficacy

%

GM06151

RT-PCR

ATXN3

23935115

ASP cm-sirna_0422

TCUGCUGCAGCUGCUGCAGAA

2'-MOE*2'-F*2'-Ome

1,20,21*2,4,6,8,10,12,14,16,18*3,5,7,9,11,13,17,19

Low efficacy

Low efficacy

%

GM06151

RT-PCR

ATXN3