aspsirna3172 details

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ASPsiRNA information siRNA Id:aspsirna3172
siRNA Name: miCAG10
Mismatch information Wild siRNA (As strand 3'->5'):GUUGCUGCUGCUGCUGCUGCUG
ASP-siRNA (As strand 3'->5'):GUUGCUGCUACUGCUGCUGCUG
Mismatch position in siRNA: A10G
Gene Information Gene Name Hungtintin (HTT)
Target Sequence (5'->3'):NA
Wild allele (5'->3'):CAACGACGACGACGACGACGAC
Mutant allele (5'->3'):CAACGACGAUGACGACGACGAC
Position of siRNA on target gene: NA
Respective Gene/Protein Resources GenBank Accession:NM_002111.7
Cytogenic location:4p16.3
Chromosomal coordinates:4:3,076,407-3,245,686
UniProt ID:P42858
HUGO ID:4851
Reference SNp(RefSNP): NA
Disease/Mutation information Target Mutation: (CAG)81
Matation type/variant: microsatellite
Mutation at gene level: NG_009378.1:g.5197CAG(36_39)
Pathogenic status of mutation: Pathogenic
Disease:Huntington disease (HD)
Clinical Resources ClinVar ID:31916
KEGG disease ID:H00059
OMIM ID: 143100
COSMIC: HTT
DECIPHER: HTT
GeneTests: HTT
ASP siRNA details Mutant allele (5'->3'): CAACGACGAUGACGACGACGAC
ASP-siRNA (As strand 3'->5'):GUUGCUGCUACUGCUGCUGCUG
Percentage efficacy of ASP-siRNA for mutant allele:80
Wild allele (5'->3'): CAACGACGACGACGACGACGAC
ASP-siRNA (As strand 3'->5'):GUUGCUGCUACUGCUGCUGCUG
Percentage efficacy of ASP-siRNA for wild allele:90
Relative difference:-10
Wild siRNA details Wild allele (5'->3'): CAACGACGACGACGACGACGAC
Wild siRNA (As strand 3'->5'):GUUGCUGCUGCUGCUGCUGCUG
Percentage efficacy of wild siRNA for wild allele:45
Wild allele (5'->3'): CAACGACGACGACGACGACGAC
ASP-siRNA (As strand 3'->5'):GUUGCUGCUACUGCUGCUGCUG
Percentage efficacy of Wild sirna for mutant allele:57
Relative difference :-12
General information Mismatch incorporated in siRNA/Target:siRNA
Cell-line used: HEK-293
Experimental technique used: Dual luciferase reporter assay
Transfection method: Lipofectamine 2000
siRNA expression method: Invitrogen
Post-transfection duration: 24 hours
Concentration used:40 nM
Reference:25849618
Delivery method:miRNA-expressing plasmids
Article title:Artificial miRNAs Targeting Mutant Huntingtin Show Preferential Silencing In Vitro and In Vivo
Authors:Monteys AM,Wilson MJ,Boudreau RL,Spengler RM,Davidson BL
Journal Reference:Mol Ther Nucleic Acids. 2015 Apr 7;4:e234. doi: 10.1038/mtna.2015.7