| ncRNA name | IMPDH1 shRNA |
| ncRNA Category | shRNA |
| Disease name | retinitis pigmentosa (RP) |
| Species | Mus musculus |
| Tissues/Cell_line | retinas |
| Methods | shRNA knockdown, qRT-PCR, histological and immunohistochemical analysis, ERG, Western blot |
| Expression pattern | associated |
| Functional description | AAV-mediated co-expression in the murine retina of a mutant human IMPDH1 gene together with shRNAs validated in vitro and in vivo, targeting both human and mouse IMPDH1, substantially suppresses the negative pathological effects of mutant IMPDH1, at a point where, in the absence of shRNA, expression of mutant protein in the RP10 model essentially ablates all photoreceptors in transfected areas of the retina. |
| PubMed ID | 18385099 |
| Year | 2008 |
| Title | Therapeutic benefit derived from RNAi-mediated ablation of IMPDH1 transcripts in a murine model of autosomal dominant retinitis pigmentosa (RP10). |
| Drug-related ncRNA | NA |
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