aspsirna3305 details

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ASPsiRNA information siRNA Id:aspsirna3305
siRNA Name: miSNP67T-4
Mismatch information Wild siRNA (As strand 3'->5'):CUCGGAAACCUUCAGACGCGG
ASP-siRNA (As strand 3'->5'):CUCGGAAACCUUCAGACACGG
Mismatch position in siRNA: A18G
Gene Information Gene Name Hungtintin (HTT)
Target Sequence (5'->3'):AGGCGGCAGCTGGGGCCGGAGCCTTTGGAAGTCTGCGCCCTTG
Wild allele (5'->3'):GAGCCUUUGGAAGUCUGCGCC
Mutant allele (5'->3'):GAGCCUUUGGAAGUCUGUGCC
Position of siRNA on target gene: 9793-9813
Respective Gene/Protein Resources GenBank Accession:NM_002111.7
Cytogenic location:4p16.3
Chromosomal coordinates:4:3,076,407-3,245,686
UniProt ID:Q96CV9
HUGO ID:4851
Reference SNp(RefSNP): rs362307
Disease/Mutation information Target Mutation: NM_002111.6:c.9810C>T
Mutation at gene level: NG_009378.1:g.170444C>T
Pathogenic status of mutation: Pathogenic
Disease:Huntington disease (HD)
Clinical Resources ClinVar ID:NA
KEGG disease ID:H00059
OMIM ID: 143100
COSMIC: HTT
DECIPHER: HTT
GeneTests: HTT
ASP siRNA details Mutant allele (5'->3'): GAGCCUUUGGAAGUCUGUGCC
ASP-siRNA (As strand 3'->5'):CUCGGAAACCUUCAGACACGG
Percentage efficacy of ASP-siRNA for mutant allele:40
Wild allele (5'->3'): GAGCCUUUGGAAGUCUGCGCC
ASP-siRNA (As strand 3'->5'):CUCGGAAACCUUCAGACACGG
Percentage efficacy of ASP-siRNA for wild allele:0
Relative difference:40
Wild siRNA details Wild allele (5'->3'): GAGCCUUUGGAAGUCUGCGCC
Wild siRNA (As strand 3'->5'):CUCGGAAACCUUCAGACGCGG
Percentage efficacy of wild siRNA for wild allele:55
Wild allele (5'->3'): GAGCCUUUGGAAGUCUGCGCC
ASP-siRNA (As strand 3'->5'):CUCGGAAACCUUCAGACACGG
Percentage efficacy of Wild sirna for mutant allele:100
Relative difference :-45
General information Mismatch incorporated in siRNA/Target:siRNA
Cell-line used: HEK293T
Experimental technique used: Dual luciferase reporter assay
Transfection method: Lipofectamine 2000
siRNA expression method: Chemically synthesized
Post-transfection duration: 48 hours
Concentration used:50 ng
Reference:27003755
Delivery method:Transfection
Article title:Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease
Authors:Miniarikova J,, Zanella I, Huseinovic A, van der Zon T, Hanemaaijer E, Martier R,, Koornneef A, Southwell AL, Hayden MR, van Deventer SJ, Petry H, Konstantinova P.
Journal Reference:Mol Ther Nucleic Acids. 2016 Mar 22;5:e297. doi: 10.1038/mtna.2016.7.