| ASPsiRNA ID | Pairing of target region (top) and ASP-siRNA (bottom) | Efficacy (mut)(%) | Efficacy (wt) (%) | Diff | Gene | Disease | Mutation | RefSNP | Analysis | Pubmed ID |
|---|---|---|---|---|---|---|---|---|---|---|
| aspsirna3143 | UUUGAGGCCUGGAUCUGUG AAACUCCGGACCUAGACAC |
0 | 9 | -9 | TP63 | Ectrodactily-ectodermal dysplasia and cleft lip/palate (EEC) | c.727C>T, p.Arg243Trp | rs12190883 | 
|
|
| aspsirna3292 | UCCCUCAUCUACUGUGUGCAC AGGGAGUAGAUGACACACGUG |
45 | 40 | 5 | HTT | Huntington disease (HD) | NM_002111.7:c.6931T>C | rs362331 |
|
|
| aspsirna3313 | GGAAGUCUGUGCCCUUGUGCC CCUUCAGACACGGGAACACGG |
30 | 10 | 20 | HTT | Huntington disease (HD) | NM_002111.6:c.9810C>T | rs362307 |
|
|
| aspsirna3164 | CAACGACGAUGACGACGACGAC GUUGCUGCUACUGCUGCUGCUG |
95 | 90 | 5 | HTT | Huntington disease (HD) | (CAG)81 | NA |
|
|
| aspsirna3168 | UUUGGAAGUCUGUGCCCUUGUA AAACCUUCAGACACGGGAACAU |
78 | 20 | 58 | HTT | Huntington disease (HD) | rs362306 | rs362307 |
|
|
| aspsirna3172 | CAACGACGAUGACGACGACGAC GUUGCUGCUACUGCUGCUGCUG |
80 | 90 | -10 | HTT | Huntington disease (HD) | (CAG)81 | NA |
|
|
| aspsirna3176 | UUUGGAAGUCUGUGCCCUUGUA AAACCUUCAGACACGGGAACAU |
80 | 20 | 60 | HTT | Huntington disease (HD) | rs362307 | rs362307 |
|
|
| aspsirna2236 | UUCAGCAGCAGUAGCAGCAGU AAGUCGUCGUCAUCGUCGUCU |
75 | 34 | 41 | ATN-1 | Dentatorubral-pallidoluysian atrophy (DRPLA) | c.1462_1464CAG(90_93) | rs19392293 |
|
|
| aspsirna3056 | UACACGGACUGCACGGAGA AUGUGCCUGACGUGCCUCU |
62 | 15 | 47 | TGFBI | Lattice corneal dystrophy type I (LCDI) | c.370C>T,p.Arg124Cys | rs12190921 |
|
|
| aspsirna0652 | CACACCCUAUUCGGCUGCC GUGUGGGAUAAGCCGACGG |
55 | 5 | 50 | KCNH2 | Long QT Syndrome (LQTS) | c.1682C>T ,p.Ala561Val | rs12191250 |
|
|
| aspsirna0666 | AAGUCCUUCUAGCAGCAGC UUCAGGAAGAUCGUCGUCG |
82 | 18 | 64 | HTT | Huntington disease (HD) | c.52CAG(36_39) | rs19392295 |
|
|
| aspsirna0585 | CUCAAUUCUUGGCUGGCCU GAGUUAAGAACCGACCGGA |
25 | 0 | 25 | KRT9 | Epidermolytic palmoplantar keratoderma (EPPK) | c.487C>T,p.Arg163Trp | rs59616921 |
|
|
| 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 |
|
|
| aspsirna0781 | CAGCAGCAGUAGCAGCAGC GUCGUCGUCAUCGUCGUCG |
50 | 40 | 10 | HTT | Huntington disease (HD) | c.52CAG(36_39) | rs19392295 |
|
|
| aspsirna0815 | UUGGAAGUCUGUGCCCUUGUA AACCUUCAGACACGGGAACAU |
65 | 15 | 50 | HTT | Huntington disease (HD) | c.52CAG(36_39) | rs362307 |
|
|
| aspsirna0049 | UCCCUCAUCUACUGUGUGC AGGGAGUAGAUGACACACG |
70 | 0 | 70 | HTT | Huntington disease (HD) | c.6931T>C,c.52CAG(36_39) | rs362331 |
|
|
| aspsirna1778 | GCUGCGCUAUGGCGAAGAC CGACGCGAUACCGCUUCUG |
NA | 60 | NA | POLR2A | Cancer | c.2907C>T,(p.Ile969=) | rs14801110 |
|
|
| aspsirna1784 | GCUGCGCUAUGGCGAAGAC CGACGCGAUACCGCUUCUG |
NA | 60 | NA | POLR2A | Cancer | c.2907C>T,(p.Ile969=) | rs14801110 |
|
|
| aspsirna0104 | CGAGUAAGCUAAAAACCAA GCUCAUUCGAUUUUUGGUU |
86 | 98 | -12 | PRNP | Prion disease/Gerstmann-Straussler disease | c.314C>T,p.Pro105Leu | rs11538758 |
|
|
| aspsirna0114 | GGAACAAGCUGAGUAAGCC CCUUGUUCGACUCAUUCGG |
85 | 90 | -5 | PRNP | Prion disease/Gerstmann-Straussler disease | c.305C>T,p.Pro102Leu | rs74315401 |
|
|
| aspsirna0143 | CGAGUAAGCUAAAAACCAA GCUCAUUCGAUUUUUGGUU |
86 | 98 | -12 | PRNP | Prion disease/Gerstmann-Straussler disease | c.314C>T,p.Pro105Leu | rs11538758 |
|
|
| aspsirna3767 | AAAGUUGCGCGGAGGAGUU UUUCAACGCACCUCCUCAA |
90 | 70 | 20 | PPIB/Cyclophilin B | PPIB-Related Osteogenesis Imperfecta (Type IX) | NA | NA |
|
|
| aspsirna0945 | AUGACCUUCUAGCAGCAGC UACUGGAAGAUCGUCGUCG |
70 | 12 | 58 | AR | Spinal and Bulbar Muscular Atrophy, X-linked 1; SMAX1/Kennedy’s disease | c.172_174CAG(10_36) (p.Gln69_Gln80del)] | rs19392293 |
|
