| | |
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| ASPsiRNA information |
siRNA Id: | aspsirna3432
|
|
siRNA Name: | miSNP50T-101
|
| Mismatch information |
Wild siRNA (As strand 3'->5'): | UAGAUGACACACGUGAAGUAG
|
|
ASP-siRNA (As strand 3'->5'): | UAGAUGACACACGUUAAGUAG
|
|
Mismatch position in siRNA: | A4G,U15G |
| Gene Information |
Gene Name | Hungtintin (HTT) |
|
Target Sequence (5'->3'): | TTGTGACCCACGCCTGCTCCCTCATCTACTGTGTGCACTTCAT
|
|
Wild allele (5'->3'): | AUCUACUGUGUGCACUUCAUC
|
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Mutant allele (5'->3'): | AUCUACUGUGUGCAAUUCAUC
|
|
Position of siRNA on target gene: | 7244-7264
|
| 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): | rs362331 |
| Disease/Mutation information |
Target Mutation: | NM_002111.7:c.6931T>C
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|
Mutation at gene level: | NG_009378.1:g.144434T>C
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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'): | AUCUACUGUGUGCAAUUCAUC
|
|
ASP-siRNA (As strand 3'->5'): | UAGAUGACACACGUUAAGUAG
|
|
Percentage efficacy of ASP-siRNA for mutant allele: | 55
|
|
Wild allele (5'->3'): | AUCUACUGUGUGCACUUCAUC
|
|
ASP-siRNA (As strand 3'->5'): | UAGAUGACACACGUUAAGUAG
|
|
Percentage efficacy of ASP-siRNA for wild allele: | 15
|
|
Relative difference: | 40
|
| Wild siRNA details |
Wild allele (5'->3'): | AUCUACUGUGUGCACUUCAUC
|
|
Wild siRNA (As strand 3'->5'): | UAGAUGACACACGUGAAGUAG
|
|
Percentage efficacy of wild siRNA for wild allele: | 50
|
|
Wild allele (5'->3'): | AUCUACUGUGUGCACUUCAUC
|
|
ASP-siRNA (As strand 3'->5'): | UAGAUGACACACGUUAAGUAG
|
|
Percentage efficacy of Wild sirna for mutant allele: | 50
|
|
Relative difference : | 0
|
| 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. |
'
