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Nonclinical Safety Evaluation of scAAV8-RLBP1 for Treatment of RLBP1 Retinitis Pigmentosa

Retinitis pigmentosa is a form of retinal degeneration usually caused by genetic mutations affecting key functional proteins. We have previously demonstrated efficacy in a mouse model of RLBP1 deficiency with a self-complementary AAV8 vector carrying the gene for human RLBP1 under control of a short...

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Detalles Bibliográficos
Autores principales:	MacLachlan, Timothy K., Milton, Mark N., Turner, Oliver, Tukov, Francis, Choi, Vivian W., Penraat, Jan, Delmotte, Marie-Hélène, Michaut, Lydia, Jaffee, Bruce D., Bigelow, Chad E.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2017
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5772508/ https://www.ncbi.nlm.nih.gov/pubmed/29359172 http://dx.doi.org/10.1016/j.omtm.2017.12.001

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