Long-term rescue of cone photoreceptor degeneration in retinitis pigmentosa 2 (RP2)-knockout mice by gene replacement therapy

Retinal neurodegenerative diseases are especially attractive targets for gene replacement therapy, which appears to be clinically effective for several monogenic diseases. X-linked forms of retinitis pigmentosa (XLRP) are relatively severe blinding disorders, resulting from progressive photoreceptor...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Hum Mol Genet
Prif Awduron: Mookherjee, Suddhasil, Hiriyanna, Suja, Kaneshiro, Kayleigh, Li, Linjing, Li, Yichao, Li, Wei, Qian, Haohua, Li, Tiansen, Khanna, Hemant, Colosi, Peter, Swaroop, Anand, Wu, Zhijian
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: Oxford University Press 2015
Pynciau:
Articles
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC4626763/
https://ncbi.nlm.nih.gov/pubmed/26358772
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddv354
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