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Gene Therapy in a Large Animal Model of PDE6A-Retinitis Pigmentosa

Despite mutations in the rod phosphodiesterase 6-alpha (PDE6A) gene being well-recognized as a cause of human retinitis pigmentosa, no definitive treatments have been developed to treat this blinding disease. We performed a trial of retinal gene augmentation in the Pde6a mutant dog using Pde6a deliv...

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Detalles Bibliográficos
Autores principales:	Mowat, Freya M., Occelli, Laurence M., Bartoe, Joshua T., Gervais, Kristen J., Bruewer, Ashlee R., Querubin, Janice, Dinculescu, Astra, Boye, Sanford L., Hauswirth, William W., Petersen-Jones, Simon M.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Frontiers Media S.A. 2017
Materias:	Neuroscience
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5476745/ https://www.ncbi.nlm.nih.gov/pubmed/28676737 http://dx.doi.org/10.3389/fnins.2017.00342

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