Total number of Results for Trangenic mouse model are 4
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
aspsirna3561 UGGAGACUUGCGCAAUGUG
ACCUCUGAACGCGUUACAC
90 10 80 SOD1 Amyotrophic Lateral Sclerosis (ALS) c.256G>C,p.Gly86Arg rs12191243
EP1567539B1
aspsirna3562 GGAGACUUGCGCAAUGUGA
CCUCUGAACGCGUUACACU
80 20 60 SOD1 Amyotrophic Lateral Sclerosis (ALS) c.256G>C,p.Gly86Arg rs12191243
EP1567539B1
aspsirna3563 GAGACUUGCGCAAUGUGAC
CUCUGAACGCGUUACACUG
96 23 73 SOD1 Amyotrophic Lateral Sclerosis (ALS) c.256G>C,p.Gly86Arg rs12191243
EP1567539B1
aspsirna3568 GACAAAGAUGCUGUGGCCGAU
CUGUUUCUACGACACCGGCUA
87 20 67 SOD1 Amyotrophic Lateral Sclerosis (ALS) c.281G>C (p.Gly94Ala) rs12191243
EP1567539B1