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siRNA Id: | aspsirna1947
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siRNA Name: | AAT-ASO
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| Gene: | SERPINA1/Alpha-1 antitrypsin (AAT) |
| Disease: | Emphysema/Alpha-1 antitrypsin deficiency (AATD) |
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Wild allele/target (5'->3'): | GACCCCAGGCTCCACCAAG
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Mutant allele/target (5'->3'): | GACCCCAGGCTCCACCAAA
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Target Mutation: | c.1096G>A,p.Glu366Lys on M2,Glu342Lys on M1A
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Wild siRNA (As strand): | CUGGGGUCCGTGGUGGUUC
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ASP-siRNA (As strand): | CUGGGGUCCGTGGUGGUUU
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Mismatch position in siRNA: | G5C |
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Mutant allele (5'->3'): | GACCCCAGGCTCCACCAAA
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ASP-siRNA (As strand): | CUGGGGUCCGTGGUGGUUU
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ASP-siRNA efficacy for mutant allele: | 80
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Wild allele (5'->3'): | GACCCCAGGCTCCACCAAG
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ASP-siRNA (As strand): | CUGGGGUCCGTGGUGGUUU
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ASP-siRNA efficacy for wild allele: | 14
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Percentage discrimination for ASP-siRNA: | 66
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Wild allele (5'->3'): | GACCCCAGGCTCCACCAAG
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Wild siRNA (As strand): | CUGGGGUCCGTGGUGGUUC
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Wild sirna efficacy for wild allele: | 22
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Wild allele (5'->3'): | GACCCCAGGCTCCACCAAG
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ASP-siRNA (As strand): | CUGGGGUCCGTGGUGGUUU
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Wild sirna efficacy for mutant allele: | 9
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Percentage discrimination for wild siRNA: | 13
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| Concentration: | 0.25microM |
| Orientation: | Double strand |
| Mismatch in: | siRNA |
| UniProt ID: | P01009 |
| Target sequence: | NA |
| Genbank Accesion: | NM_001127701.1 |
| Cell-line: | PiZ hepatocytes |
| Experimental technique used: | RT-PCR |
| Transfection method: | Electroporation |
| siRNA expression method: | Applied Biosystems |
| Post-transfection duration: | 24 hours |
| OMID ID: | 613490 |
| KEGG disease ID: | H01103 |
| Reference: | 24355919 |
