| ASPsiRNA ID | Pairing of target region (top) and ASP-siRNA (bottom) | Efficacy (mut)(%) | Efficacy (wt) (%) | Diff | Gene | Disease | Mutation | RefSNP | Analysis | Pubmed ID |
|---|---|---|---|---|---|---|---|---|---|---|
| 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 | 
|
|
| 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 |
|
|
| 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 |
|
|
| 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 |
|
