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siRNA Id: | aspsirna0347
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siRNA Name: | ShAtaxMUT (C)
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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'): | AGCAGCAGGGGGACCUAUC
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Mutant allele/target (5'->3'): | AGCAGCAGCGGGACCUAUC
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Target Mutation: | c.892_894CAG(8_36) (p.Gln298_Gln305=),c.987G>C
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Wild siRNA (As strand): | UCGUCGUCCCCCUGGAUAG
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ASP-siRNA (As strand): | UCGUCGUCGCCCUGGAUAG
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Mismatch position in siRNA: | G11C |
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Mutant allele (5'->3'): | AGCAGCAGCGGGACCUAUC
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ASP-siRNA (As strand): | UCGUCGUCGCCCUGGAUAG
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ASP-siRNA efficacy for mutant allele: | 16
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Wild allele (5'->3'): | AGCAGCAGGGGGACCUAUC
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ASP-siRNA (As strand): | UCGUCGUCGCCCUGGAUAG
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ASP-siRNA efficacy for wild allele: | 0
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Percentage discrimination for ASP-siRNA: | 16
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Wild allele (5'->3'): | AGCAGCAGGGGGACCUAUC
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Wild siRNA (As strand): | UCGUCGUCCCCCUGGAUAG
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Wild sirna efficacy for wild allele: | 17
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Wild allele (5'->3'): | AGCAGCAGGGGGACCUAUC
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ASP-siRNA (As strand): | UCGUCGUCGCCCUGGAUAG
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Wild sirna efficacy for mutant allele: | 20
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Percentage discrimination for wild siRNA: | -3
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| Concentration: | 5mg |
| Orientation: | Sense |
| Mismatch in: | siRNA |
| UniProt ID: | P54252 |
| Target sequence: | GCAGCAGCAGCAGCAGCAGCAGGGGGACCUAUC |
| Genbank Accesion: | NM_004993.5 |
| Cell-line: | 293T |
| Experimental technique used: | Western blot, RT-PCR |
| Transfection method: | CaPO4 mediated transfection |
| siRNA expression method: | Chemically synthesized |
| Post-transfection duration: | 48 hours |
| OMID ID: | 109150 |
| KEGG disease ID: | H00063 |
| Reference: | 18841197 |
