| | |
ASPsiRNA information |
siRNA Id: | aspsirna3426
|
siRNA Name: | MIH12_451
|
Mismatch information |
Wild siRNA (As strand 3'->5'): | NA
|
ASP-siRNA (As strand 3'->5'): | GAAGCUCAGGGAGUUCAGGAA
|
Mismatch position in siRNA: | NA |
Gene Information |
Gene Name | Hungtintin (HTT) |
Target Sequence (5'->3'): | CAGGUUCUACCUGCCGGCGAGUCCAAGACGAAAAUGGACGCCGGGUCUCGGGGUAAGUAACGGGGCCACGACUCGCCGCGGCGCUCAGC
|
Wild allele (5'->3'): | NA
|
Mutant allele (5'->3'): | CUUCGAGUCCCUCAAGUCCUU
|
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: | Q96CV9 |
HUGO ID: | 4851 |
Reference SNp(RefSNP): | NA |
Disease/Mutation information |
Target Mutation: | (CAG)n
|
Mutation at gene level: | NA
|
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'): | CUUCGAGUCCCUCAAGUCCUU
|
ASP-siRNA (As strand 3'->5'): | GAAGCUCAGGGAGUUCAGGAA
|
Percentage efficacy of ASP-siRNA for mutant allele: | 32
|
Wild allele (5'->3'): | NA
|
ASP-siRNA (As strand 3'->5'): | GAAGCUCAGGGAGUUCAGGAA
|
Percentage efficacy of ASP-siRNA for wild allele: | 0
|
Relative difference: | 32
|
Wild siRNA details |
Wild allele (5'->3'): | NA
|
Wild siRNA (As strand 3'->5'): | NA
|
Percentage efficacy of wild siRNA for wild allele: | NA
|
Wild allele (5'->3'): | NA
|
ASP-siRNA (As strand 3'->5'): | GAAGCUCAGGGAGUUCAGGAA
|
Percentage efficacy of Wild sirna for mutant allele: | NA
|
Relative difference : | NA
|
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: | AAV5 vector mediated delivery |
'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. |
'