Codon-Optimized RPGR Improves Stability and Efficacy of AAV8 Gene Therapy in Two Mouse Models of X-Linked Retinitis Pigmentosa

X-linked retinitis pigmentosa (XLRP) is generally a severe form of retinitis pigmentosa, a neurodegenerative, blinding disorder of the retina. 70% of XLRP cases are due to mutations in the retina-specific isoform of the gene encoding retinitis pigmentosa GTPase regulator (RPGR(ORF15)). Despite succe...

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Detalles Bibliográficos
Autores principales: Fischer, M. Dominik, McClements, Michelle E., Martinez-Fernandez de la Camara, Cristina, Bellingrath, Julia-Sophia, Dauletbekov, Daniyar, Ramsden, Simon C., Hickey, Doron G., Barnard, Alun R., MacLaren, Robert E.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2017
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5542800/
https://www.ncbi.nlm.nih.gov/pubmed/28549772
http://dx.doi.org/10.1016/j.ymthe.2017.05.005

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5542800/
https://www.ncbi.nlm.nih.gov/pubmed/28549772
http://dx.doi.org/10.1016/j.ymthe.2017.05.005

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