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Gene therapy provides long-term visual function in a pre-clinical model of retinitis pigmentosa

Approximately 36 000 cases of simplex and familial retinitis pigmentosa (RP) worldwide are caused by a loss in phosphodiesterase (PDE6) function. In the preclinical Pde6α(nmf363) mouse model of this disease, defects in the α-subunit of PDE6 result in a progressive loss of photoreceptors and neuronal...

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Autors principals: Wert, Katherine J., Davis, Richard J., Sancho-Pelluz, Javier, Nishina, Patsy M., Tsang, Stephen H.
Format: Artigo
Idioma:Inglês
Publicat: Oxford University Press 2013
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Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3542865/
https://ncbi.nlm.nih.gov/pubmed/23108158
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/dds466
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