| ASPsiRNA ID | Pairing of target region (top) and ASP-siRNA (bottom) | Efficacy (mut)(%) | Efficacy (wt) (%) | Diff | Gene | Disease | Mutation | RefSNP | Analysis | Pubmed ID |
|---|---|---|---|---|---|---|---|---|---|---|
| aspsirna3570 | GUGGCUCACCAGAUUACAC CACCGAGUGGUCUAAUGUG |
45 | 10 | 35 | ACVR1 | Fibrodysplasia Ossificans Progressiva (FOP) | c.617G>A,p.Arg206His | rs12191267 | 
|
|
| aspsirna2016 | UUUAUGAAGAUCUCUCUGA AAAUACUUCUAGAGAGACU |
82 | 30 | 52 | P. luciferase | NA | NA | NA |
|
|
| aspsirna2073 | CUCUCCUAUAGCUAAGGCC GAGAGGAUAUCGAUUCCGG |
45 | 45 | 0 | PPIB/Cyclophilin B | NA | NA | NA |
|
|
| aspsirna3192 | UUUCGACAUGUCCUUCCAG AAAGCUGUACAGGAAGGUC |
88 | 40 | 48 | TP63 | Ectrodactyly-Ectodermal Dysplasia-Clefting syndrome 3 (EEC3) | p.Arg132His,c.394G>A | rs19392114 |
|
|
| aspsirna3080 | GAGGUGCAUGUUUGUGCCU CUCCACGUACAAACACGGA |
75 | 20 | 55 | TP53 | Cancer | NC_000005.10:g.144594367C>A | rs57315468 |
|
|
| aspsirna3081 | UGAGGUGCAUGUUUGUGCCU ACUCCACGUACAAACACGGA |
45 | 40 | 5 | TP53 | Cancer | NC_000005.10:g.144594367C>A | rs57315468 |
|
|
| aspsirna3086 | GAGGUGCAUGUUUGUGCCU CUCCACGUACAAACACGGA |
78 | 20 | 58 | TP53 | Cancer | NC_000005.10:g.144594367C>A | rs57315468 |
|
|
| aspsirna3087 | UGAGGUGCAUGUUUGUGCCU ACUCCACGUACAAACACGGA |
40 | 40 | 0 | TP53 | Cancer | NC_000005.10:g.144594367C>A | rs57315468 |
|
|
| aspsirna3092 | GAGGUGCAUGUUUGUGCCU CUCCACGUACAAACACGGA |
70 | 20 | 50 | TP53 | Cancer | NC_000005.10:g.144594367C>A | rs57315468 |
|
|
| aspsirna3093 | UGAGGUGCAUGUUUGUGCCU ACUCCACGUACAAACACGGA |
45 | 40 | 5 | TP53 | Cancer | NC_000005.10:g.144594367C>A | rs57315468 |
|
|
| aspsirna2181 | AGAAGCCAGAGAUCCUGGA UCUUCGGUCUCUAGGACCU |
80 | 40 | 40 | COL6A1 | Ullrich congenital muscular dystrophy (UCMD),Bethlem myopathy (BM) | c.850G>A, p.G284R | rs1219 |
|
|
| aspsirna1905 | GUCACCCUCAGGUGAGGGUGG CAGUGGGAGUCCACUCCCACC |
Moderate efficacy | Moderate efficacy | Poor discrimination | Myh6 | Human hypertrophic cardiomyopathy (HCM) | c.2384G>A,p.Arg795Gln | rs26760690 |
|
|
| aspsirna0606 | AAUUCUCAGCUGGCCUCUU UUAAGAGUCGACCGGAGAA |
39 | 20 | 19 | KRT9 | Epidermolytic palmoplantar keratoderma (EPPK) | c.488G>A,p.Arg163Gln | rs57758262 |
|
|
| aspsirna0323 | CUGGUAUUCCCAGGCUCCC GACCAUAAGGGUCCGAGGG |
32 | 32 | 0 | COL7A1 | Dominant dystrophic epidermolysis bullosa(DDEB) | c.6127G>A,p.Gly2043Arg | rs12191283 |
|
|
| aspsirna0338 | UAUUCCCAGGCUCCCAGGC AUAAGGGUCCGAGGGUCCG |
45 | 5 | 40 | COL7A1 | Dominant dystrophic epidermolysis bullosa(DDEB) | c.6127G>A,p.Gly2043Arg | rs12191283 |
|
|
| aspsirna0252 | UGACUACAGCAUUGCUCAG ACUGAUGUCGUAACGAGUC |
80 | 65 | 15 | LRRK2 | Parkinsons disease (PD) | c.6055G>A,p.Gly2019Ser | rs34637584 |
|
|
| aspsirna3122 | CCCUCAAAAACAAGUUUGC GGGAGUUUUUGUUCAAACG |
35 | 0 | 35 | KRT6A | Pachyonychia congenita (PC) | c.513C>A,p.Asn171Lys | rs59685571 |
|
|
| aspsirna3123 | CCCUCAAAAACAAGUUUGC GGGAGUUUUUGUUCAAACG |
55 | 10 | 45 | KRT6A | Pachyonychia congenita (PC) | c.513C>A,p.Asn171Lys | rs59685571 |
|
|
| aspsirna0858 | GCCAGCCAUGAACAAUGUC CGGUCGGUACUUGUUACAG |
83 | 30 | 53 | ATXN7 | Spino Cerebellar Ataxia type-7 (SCA-7) | c.88_90CAG(7_17) | rs193922929 |
|
|
| aspsirna0065 | AGUGAUCAUCAUCACCUUG UCACUAGUAGUAGUGGAAC |
3 | 17 | -14 | APP | Alzheimers disease | c.2010_2011delGAinsTC (p.Lys670_Met671delinsAsnLeu) | rs28186516 |
|
|
| aspsirna0090 | UGUGCACAACUGCGUCAAU ACACGUGUUGACGCAGUUA |
88 | 70 | 18 | PRNP | Prion disease/Creutzfeldt-Jakob disease | c.532G>A,p.Asp178Asn | rs74315403 |
|
|
| aspsirna3505 | UGUGCACAACUGCGUCAAU ACACGUGUUGACGCAGUUA |
88 | 70 | 18 | PRNP | Prion disease/Creutzfeldt-Jakob disease | c.532G>A,p.Asp178Asn | rs74315403 |
|
|
| aspsirna0887 | CCCUCAAAAACAAGUUUGC GGGAGUUUUUGUUCAAACG |
35 | 0 | 35 | KRT6a | Pachyonychia congenita (PC) | c.513C>A,p.Asn171Lys | rs59685571 |
|
|
| aspsirna3624 | UCAGAGAGAUCCUCAUAAA AGUCUCUUUAGGAGUAUUU |
90 | 82 | 8 | PPIB/Cyclophilin B | PPIB-Related Osteogenesis Imperfecta (Type IX) | NA | NA |
|
|
| aspsirna3700 | GGUUGGAGGAGAGAUGAUA CCAACCUUCUCUCUACUAU |
90 | 80 | 10 | PPIB/Cyclophilin B | PPIB-Related Osteogenesis Imperfecta (Type IX) | NA | NA |
|
|
| aspsirna0526 | CAUGGUGUGACAACAGUGGCU GUACCACACUGUUGUCACCGA |
45 | 70 | -25 | SNCA | Parkinsons disease (PD) | c.157G>A,p.Ala53Thr | rs10489387 |
|
|
| aspsirna0535 | CAUGGUGUGACAACAGUGGCU GUACCACACUGUUGUCACCGA |
45 | 70 | -25 | SNCA | Parkinsons disease (PD) | c.157G>A,p.Ala53Thr | rs10489387 |
|
|
| aspsirna0570 | ACAGUGAUCAUCAUCACCUUG UGUCACUAGUAGUAGUGGAAC |
73 | 68 | 5 | APP | Alzheimers disease | c.2149G>A,p.Val717Ile | rs63750264 |
|
|
| aspsirna0573 | CGCGACAGUGAUCAUCAUCACCUUG GCGCUGUCACUAGUAGUAGUGGAAC |
20 | 58 | -38 | APP | Alzheimers disease | c.2149G>A,p.Val717Ile | rs63750264 |
|
|
| aspsirna1791 | UGUGGCCAUGCAUGUGUUC ACACCGGUACGUACACAAG |
NA | 5 | NA | TTR | Amyloidogenic transthyretin amyloidosis | c.148G>A,p.Val50Met | rs28933979 |
|
|
| aspsirna0199 | GUGGCCAGGUAGAAGUAAAAUC CACCGGUCCAUCUUCAUUUUAG |
86 | Poor efficacy | High discrimination | MAPT | Frontotemporal dementia and parkinsonism linked to chromosome 17 (FTDP-17) | c.1960G>A,p.Val654Met | rs63750570 |
|
