aspsirna3032 details

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ASPsiRNA information siRNA Id:aspsirna3032
siRNA Name: P9
Mismatch information Wild siRNA (As strand 3'->5'):GUCGUCGUCGUCGUCGUCG
ASP-siRNA (As strand 3'->5'):GUCGUCGUCGACGUCGUCG
Mismatch position in siRNA: A9U
Gene Information Gene Name Ataxin-3 (ATXN3)
Target Sequence (5'->3'):CAGCAGCAGCAGCAGCAGCAGCAGGGGGACCTATCAGGACAGAGTT
Wild allele (5'->3'):CAGCAGCAGCAGCAGCAGC
Mutant allele (5'->3'):CAGCAGCAGCUGCAGCAGC
Position of siRNA on target gene: 961-979
Respective Gene/Protein Resources GenBank Accession:NM_004993.5
Cytogenic location:14q32.12  
Chromosomal coordinates:14:92,524,895-92,572,964
UniProt ID:P54252
HUGO ID:7106
Reference SNp(RefSNP): rs19392292
Disease/Mutation information Target Mutation: c.892_894CAG(8_36) (p.Gln298_Gln305=)
Matation type/variant: Microsatellite/synonymous variant
Mutation at gene level: NG_008198.2:g.40588_40590CAG(8_36)
Pathogenic status of mutation: Pathogenic
Disease:Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3)
Clinical Resources ClinVar ID:3551
KEGG disease ID:H00063
OMIM ID: 109150
COSMIC: ATXN3
DECIPHER: ATXN3
GeneTests: ATXN3
ASP siRNA details Mutant allele (5'->3'): CAGCAGCAGCUGCAGCAGC
ASP-siRNA (As strand 3'->5'):GUCGUCGUCGACGUCGUCG
Percentage efficacy of ASP-siRNA for mutant allele:80
Wild allele (5'->3'): CAGCAGCAGCAGCAGCAGC
ASP-siRNA (As strand 3'->5'):GUCGUCGUCGACGUCGUCG
Percentage efficacy of ASP-siRNA for wild allele:25
Relative difference:55
Wild siRNA details Wild allele (5'->3'): CAGCAGCAGCAGCAGCAGC
Wild siRNA (As strand 3'->5'):GUCGUCGUCGUCGUCGUCG
Percentage efficacy of wild siRNA for wild allele:70
Wild allele (5'->3'): CAGCAGCAGCAGCAGCAGC
ASP-siRNA (As strand 3'->5'):GUCGUCGUCGACGUCGUCG
Percentage efficacy of Wild sirna for mutant allele:50
Relative difference :20
General information Mismatch incorporated in siRNA/Target:siRNA
Cell-line used: Fibroblast
Experimental technique used: Western blot
Transfection method: RNAiMAX
siRNA expression method: Integrated DNA Technologies
Post-transfection duration: 24 hours
Concentration used:12nM
Reference:21294677
Delivery method:Transfection
Article title:Allele-selective inhibition of ataxin-3 (ATX3) expression by antisense oligomers and duplex RNAs
Authors:Hu J, Gagnon KT, Liu J, Watts JK, Syeda-Nawaz J, Bennett CF, Swayze EE, Randolph J, Chattopadhyaya J, Corey DR
Journal Reference:Biol Chem. 2011 Apr;392(4):315-25. doi: 10.1515/BC.2011.045. Epub 2011 Feb 7