Photoreceptor Rescue by an Abbreviated Human RPGR Gene in a Murine Model of X-linked Retinitis Pigmentosa

The X-linked RP3 gene codes for the ciliary protein RPGR and accounts for over 10% of inherited retinal degenerations. The critical RPGR-ORF15 splice variant contains a highly repetitive purine-rich linker region that renders it unstable and difficult to adapt for gene therapy. To test the hypothesi...

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Pubblicato in:Gene Ther
Autori principali: Pawlyk, Basil S., Adamian, Michael, Sun, Xun, Bulgakov, Oleg V., Shu, Xinhua, Smith, Alexander J., Berson, Eliot L., Ali, Robin R., Khani, Shahrokh, F.Wright, Alan, Sandberg, Michael A., Li, Tiansen
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2015
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4863462/
https://ncbi.nlm.nih.gov/pubmed/26348595
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2015.93
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