| |
|---|
|
siRNA Id: | aspsirna3046
|
|
siRNA Name: | PM4
|
| Gene: | Ataxin-3 (ATXN3) |
| Disease: | Machado-Joseph Disease (MJD)/Spinocerebellar ataxia type 3 (SCA3) |
|
Wild allele/target (5'->3'): | CAGCAGCAGCAUCAGCAGC
|
|
Mutant allele/target (5'->3'): | CAGCAGCAUUUUCAGCAGC
|
|
Target Mutation: | c.892_894CAG(8_36) (p.Gln298_Gln305=)
|
|
Wild siRNA (As strand): | GUCGUCGUCGUAGUCGUCG
|
|
ASP-siRNA (As strand): | GUCGUCGUAAAAGUCGUCG
|
|
Mismatch position in siRNA: | A9U, A10G, A11C, A12U |
|
Mutant allele (5'->3'): | CAGCAGCAUUUUCAGCAGC
|
|
ASP-siRNA (As strand): | GUCGUCGUAAAAGUCGUCG
|
|
ASP-siRNA efficacy for mutant allele: | 75
|
|
Wild allele (5'->3'): | CAGCAGCAGCAUCAGCAGC
|
|
ASP-siRNA (As strand): | GUCGUCGUAAAAGUCGUCG
|
|
ASP-siRNA efficacy for wild allele: | 35
|
|
Percentage discrimination for ASP-siRNA: | 40
|
|
Wild allele (5'->3'): | CAGCAGCAGCAUCAGCAGC
|
|
Wild siRNA (As strand): | GUCGUCGUCGUAGUCGUCG
|
|
Wild sirna efficacy for wild allele: | 70
|
|
Wild allele (5'->3'): | CAGCAGCAGCAUCAGCAGC
|
|
ASP-siRNA (As strand): | GUCGUCGUAAAAGUCGUCG
|
|
Wild sirna efficacy for mutant allele: | 50
|
|
Percentage discrimination for wild siRNA: | 20
|
| Concentration: | 12nM |
| Orientation: | Antisense |
| Mismatch in: | siRNA |
| UniProt ID: | P54252 |
| Target sequence: | CAGCAGCAGCAGCAGCAGCAGCAGGGGGACCTATCAGGACAGAGTT |
| Genbank Accesion: | NM_004993.5 |
| Cell-line: | Fibroblast |
| Experimental technique used: | RT-PCR |
| Transfection method: | RNAiMAX |
| siRNA expression method: | Integrated DNA Technologies |
| Post-transfection duration: | 24 hours |
| OMID ID: | 109150 |
| KEGG disease ID: | H00063 |
| Reference: | 21294677 |
