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Mid-stage intervention achieves similar efficacy as conventional early-stage treatment using gene therapy in a pre-clinical model of retinitis pigmentosa

Deficiencies in rod-specific cyclic guanosine monophosphate (cGMP) phosphodiesterase-6 (PDE6) are the third most common cause of autosomal recessive retinitis pigmentosa (RP). Previously, viral gene therapy approaches on pre-clinical models with mutations in PDE6 have demonstrated that the photorece...

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Autors principals: Wert, Katherine J., Sancho-Pelluz, Javier, Tsang, Stephen H.
Format: Artigo
Idioma:Inglês
Publicat: Oxford University Press 2014
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Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3869365/
https://ncbi.nlm.nih.gov/pubmed/24101599
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddt452
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