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