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siRNA Id: | aspsirna3041
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siRNA Name: | P16
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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'): | CAGCAGCAGCAGCAGCAGC
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Mutant allele/target (5'->3'): | CAGUAGCAGCAGCAGCAGC
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Target Mutation: | c.892_894CAG(8_36) (p.Gln298_Gln305=)
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Wild siRNA (As strand): | GUCGUCGUCGUCGUCGUCG
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ASP-siRNA (As strand): | GUCAUCGUCGUCGUCGUCG
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Mismatch position in siRNA: | A16G |
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Mutant allele (5'->3'): | CAGUAGCAGCAGCAGCAGC
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ASP-siRNA (As strand): | GUCAUCGUCGUCGUCGUCG
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ASP-siRNA efficacy for mutant allele: | 80
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Wild allele (5'->3'): | CAGCAGCAGCAGCAGCAGC
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ASP-siRNA (As strand): | GUCAUCGUCGUCGUCGUCG
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ASP-siRNA efficacy for wild allele: | 60
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Percentage discrimination for ASP-siRNA: | 20
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Wild allele (5'->3'): | CAGCAGCAGCAGCAGCAGC
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Wild siRNA (As strand): | GUCGUCGUCGUCGUCGUCG
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Wild sirna efficacy for wild allele: | 70
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Wild allele (5'->3'): | CAGCAGCAGCAGCAGCAGC
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ASP-siRNA (As strand): | GUCAUCGUCGUCGUCGUCG
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Wild sirna efficacy for mutant allele: | 50
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Percentage discrimination for wild siRNA: | 20
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| Concentration: | 12nM |
| Orientation: | Antisense |
| Mismatch in: | siRNA |
| UniProt ID: | P54252 |
| Target sequence: | CAGCAGCAGCAGCAGCAGCAGCAGGGGGACCTATCAGGACAGAGTT |
| Genbank Accesion: | NM_004993.5 |
| Cell-line: | Fibroblast |
| Experimental technique used: | Western blot |
| Transfection method: | RNAiMAX |
| siRNA expression method: | Integrated DNA Technologies |
| Post-transfection duration: | 24 hours |
| OMID ID: | 109150 |
| KEGG disease ID: | H00063 |
| Reference: | 21294677 |
