Total number of Results for Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) are 44
ASPsiRNA ID Pairing of target region (top) and ASP-siRNA (bottom) Efficacy (mut)(%) Efficacy (wt) (%) Diff Gene Disease Mutation RefSNP Analysis Pubmed ID
ASPsiRNA ID Mutant allele/ASP-siRNA Efficacy
for mutant(%)		 Efficacy
for wild (%)		 Relative Difference (%) Gene Disease Mutation RefSNP Analysis Pubmed ID
aspsirna0201 CAGCAGCAGCGGGACCUAUC
GUCGUCGUCGCCCUGGAUAG		 66 62 4 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.G987C,c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
12782788
aspsirna0202 CAGCAGCCGGACCUAUCAGGAC
GUCGUCGGCCUGGAUAGUCCUG		 80 20 60 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.G987C,c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
12782788
aspsirna0203 CAGCAGCGGGACCUAUCAGGAC
GUCGUCGCCCUGGAUAGUCCUG		 55 0 55 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.G987C,c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
12782788
aspsirna0204 CAGCAGCAGGGGGACCUAUC
GUCGUCGUCCCCCUGGAUAG		 40 22 18 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.G987C,c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
12782788
aspsirna0205 UUGAAAAACAGCAGCAAAAGC
AACUUUUUGUCGUCGUUUUCG		 65 20 45 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.G987C,c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
12782788
aspsirna0206 CAGCAGCAGCAGCAGCAGCAGC
GUCGUCGUCGUCGUCGUCGUCG		 46 NA NA ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.G987C,c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
12782788
aspsirna0347 AGCAGCAGCGGGACCUAUC
UCGUCGUCGCCCUGGAUAG		 16 0 16 ATXN3 Machado-Joseph disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.892_894CAG(8_36) (p.Gln298_Gln305=),c.987G>C rs19392292
18841197
aspsirna0656 GAUAGGUCCCGCUGCUGCU
CUAUCCAGGGCGACGACGA		 25 50 -25 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.892_894CAG(8_36),(p.Gln298_Gln305=) rs19392292
23349684
aspsirna0928 GCAGCGGGACCUAUCAGGA
CGUCGCCCUGGAUAGUCCU		 52 94 -42 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.209_211CAG(8_36),(p.Gln298_Gln305=) rs19392292
15236410
aspsirna0929 GCAGCAGCGGGACCUAUCA
CGUCGUCGCCCUGGAUAGU		 28 90 -62 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.209_211CAG(8_36),(p.Gln298_Gln305=) rs19392292
15236410
aspsirna0930 GCAGCAGCAGCGGGACCUA
CGUCGUCGUCGCCCUGGAU		 89 50 39 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.209_211CAG(8_36),(p.Gln298_Gln305=) rs19392292
15236410
aspsirna0931 GCAGCAGCAGCAGCGGGAC
CGUCGUCGUCGUCGCCCUG		 80 80 0 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.209_211CAG(8_36),(p.Gln298_Gln305=) rs19392292
15236410
aspsirna3027 CAGCAGCAGCAGCAGUAGC
GUCGUCGUCGUCGUCAUCG		 60 40 20 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
21294677
aspsirna3028 CAGCAGCAGCAGCAUCAGC
GUCGUCGUCGUCGUAGUCG		 60 50 10 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
21294677
aspsirna3029 CAGCAGCAGCAGCUGCAGC
GUCGUCGUCGUCGACGUCG		 35 30 5 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
21294677
aspsirna3030 CAGCAGCAGCAGUAGCAGC
GUCGUCGUCGUCAUCGUCG		 80 70 10 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
21294677
aspsirna3031 CAGCAGCAGCAUCAGCAGC
GUCGUCGUCGUAGUCGUCG		 70 30 40 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
21294677
aspsirna3032 CAGCAGCAGCUGCAGCAGC
GUCGUCGUCGACGUCGUCG		 80 25 55 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
21294677
aspsirna3033 CAGCAGCAGUAGCAGCAGC
GUCGUCGUCAUCGUCGUCG		 80 40 40 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
21294677
aspsirna3034 CAGCAGCAGAAGCAGCAGC
GUCGUCGUCUUCGUCGUCG		 87 25 62 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
21294677
aspsirna3035 CAGCAGCAGUUGCAGCAGC
GUCGUCGUCAACGUCGUCG		 90 20 70 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
21294677
aspsirna3036 CAGCAGCAUUUGCAGCAGC
GUCGUCGUAAACGUCGUCG		 90 20 70 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
21294677
aspsirna3037 CAGCAGCAUUUUCAGCAGC
GUCGUCGUAAAAGUCGUCG		 80 30 50 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
21294677
aspsirna3038 CAGCAGCAUCAGCAGCAGC
GUCGUCGUAGUCGUCGUCG		 85 30 55 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
21294677
aspsirna3039 CAGCAGCUGCAGCAGCAGC
GUCGUCGACGUCGUCGUCG		 90 50 40 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
21294677
aspsirna3040 CAGCAGUAGCAGCAGCAGC
GUCGUCAUCGUCGUCGUCG		 70 60 10 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
21294677
aspsirna3041 CAGUAGCAGCAGCAGCAGC
GUCAUCGUCGUCGUCGUCG		 80 60 20 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
21294677
aspsirna3042 CAACAGUAGCAACAGCUGC
GUUGUCAUCGUUGUCGACG		 87 78 9 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
21294677
aspsirna3043 CAGCAGCAGCUGCAGCAGC
GUCGUCGUCGACGUCGUCG		 78 20 58 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
21294677
aspsirna3044 CAGCAGCAGUUGCAGCAGC
GUCGUCGUCAACGUCGUCG		 85 20 65 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
21294677
aspsirna3045 CAGCAGCAUUUGCAGCAGC
GUCGUCGUAAACGUCGUCG		 85 30 55 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
21294677
aspsirna3046 CAGCAGCAUUUUCAGCAGC
GUCGUCGUAAAAGUCGUCG		 75 35 40 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
21294677
aspsirna3528 CAGCAGCAGCGGGACCUAUC
GUCGUCGUCGCCCUGGAUAG		 66 62 4 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.G987C,c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
US20080176812A1
aspsirna3529 CAGCAGCCGGACCUAUCAGGAC
GUCGUCGGCCUGGAUAGUCCUG		 80 20 60 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.G987C,c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
US20080176812A1
aspsirna3530 CAGCAGCGGGACCUAUCAGGAC
GUCGUCGCCCUGGAUAGUCCUG		 55 0 55 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.G987C,c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
US20080176812A1
aspsirna3531 CAGCAGCAGGGGGACCUAUC
GUCGUCGUCCCCCUGGAUAG		 40 22 18 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.G987C,c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
US20080176812A1
aspsirna3532 UUGAAAAACAGCAGCAAAAGC
AACUUUUUGUCGUCGUUUUCG		 65 20 45 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.G987C,c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
US20080176812A1
aspsirna3533 CAGCAGCAGCAGCAGCAGCAGC
GUCGUCGUCGUCGUCGUCGUCG		 46 NA NA ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.G987C,c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
US20080176812A1
aspsirna3540 CAGCAGCAGCGGGACCUAUC
GUCGUCGUCGCCCUGGAUAG		 66 62 4 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.G987C,c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
US20080176812A1
aspsirna3541 CAGCAGCCGGACCUAUCAGGAC
GUCGUCGGCCUGGAUAGUCCUG		 80 20 60 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.G987C,c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
US20080176812A1
aspsirna3542 CAGCAGCGGGACCUAUCAGGAC
GUCGUCGCCCUGGAUAGUCCUG		 55 0 55 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.G987C,c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
US20080176812A1
aspsirna3543 CAGCAGCAGGGGGACCUAUC
GUCGUCGUCCCCCUGGAUAG		 40 22 18 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.G987C,c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
US20080176812A1
aspsirna3544 UUGAAAAACAGCAGCAAAAGC
AACUUUUUGUCGUCGUUUUCG		 65 20 45 ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.G987C,c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
US20080176812A1
aspsirna3545 CAGCAGCAGCAGCAGCAGCAGC
GUCGUCGUCGUCGUCGUCGUCG		 46 NA NA ATXN3 Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) c.G987C,c.892_894CAG(8_36) (p.Gln298_Gln305=) rs19392292
US20080176812A1