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siRNA Id: | aspsirna0656
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siRNA Name: | ShAtx-3
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| Gene: | Ataxin-3 (ATXN3) |
| Disease: | Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) |
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Wild allele/target (5'->3'): | GAUAGGUCCCCCUGCUGCU
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Mutant allele/target (5'->3'): | GAUAGGUCCCGCUGCUGCU
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Target Mutation: | c.892_894CAG(8_36),(p.Gln298_Gln305=)
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Wild siRNA (As strand): | CUAUCCAGGGGGACGACGA
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ASP-siRNA (As strand): | CUAUCCAGGGCGACGACGA
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Mismatch position in siRNA: | C9G |
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Mutant allele (5'->3'): | GAUAGGUCCCGCUGCUGCU
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ASP-siRNA (As strand): | CUAUCCAGGGCGACGACGA
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ASP-siRNA efficacy for mutant allele: | 25
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Wild allele (5'->3'): | GAUAGGUCCCCCUGCUGCU
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ASP-siRNA (As strand): | CUAUCCAGGGCGACGACGA
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ASP-siRNA efficacy for wild allele: | 50
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Percentage discrimination for ASP-siRNA: | -25
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Wild allele (5'->3'): | GAUAGGUCCCCCUGCUGCU
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Wild siRNA (As strand): | CUAUCCAGGGGGACGACGA
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Wild sirna efficacy for wild allele: | NA
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Wild allele (5'->3'): | GAUAGGUCCCCCUGCUGCU
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ASP-siRNA (As strand): | CUAUCCAGGGCGACGACGA
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Wild sirna efficacy for mutant allele: | NA
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Percentage discrimination for wild siRNA: | NA
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| Concentration: | 6microM |
| Orientation: | Sense |
| Mismatch in: | siRNA |
| UniProt ID: | P54252 |
| Target sequence: | CAGCAAAAGCAGCAACAGCAGCAGCAGCAGCAGCA |
| Genbank Accesion: | NM_004993.5 |
| Cell-line: | Cerebellum of C57/BL6 mice |
| Experimental technique used: | Western blot |
| Transfection method: | Lipofectamine 2,000 |
| siRNA expression method: | Invitrogen |
| Post-transfection duration: | 10 weeks post injection period |
| OMID ID: | 109150 |
| KEGG disease ID: | H00063 |
| Reference: | 23349684 |
