SM2642 Chemically modified siRNA information

Field Sense Antisense
siRNA chemical modification 2-Fluoro* Phosphorothioate 2-Fluoro* Phosphorothioate
siRNA modification types 2 2
Overall number of modifications 11 11
Position of modifications 4,6,19,20 * 1,2,3,17,18,19,20 2,3,4,15,17,18 * 16,17,18,19,20
Modification on sugar or base or phosphate Sugar; Phosphate Sugar; Phosphate
SMILES
(Click to view structure & nomenclature)
OCC1OCC(F)C1O
OCC1OCC(O)C1OP(O)(S)=O


OCC1OCC(F)C1O
OCC1OCC(O)C1OP(O)(S)=O


siRNA Sequence CGAUGUGUCUAUUGAAGAUUC AUCUUCAAUAGACACAUCGGC
siRNA length base-pair 21 21
siRNA name in paper R1 R1
Biological activity 66 percent target mRNA inhibition
Experiment used to check activity Dual luciferase reporter assay
Melting temperature (oC) NA
Target gene Human SOD1 gene
siRNA concentration 2 nM
Cell or Organism used HEK293 cells and Transgenic mice FVB/NJ background (SOD1-G93A)
Transfection method Lipofectamine 2000
Duration after transfection 24 Hours
Article title Therapeutic gene silencing delivered by a chemically modified small interfering RNA against mutant SOD1 slows amyotrophic lateral sclerosis progression
Reference18367449