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New MiniPromoter Ple345 (NEFL) Drives Strong and Specific Expression in Retinal Ganglion Cells of Mouse and Primate Retina

Retinal gene therapy is leading the neurological gene therapy field, with 32 ongoing clinical trials of recombinant adeno-associated virus (rAAV)–based therapies. Importantly, over 50% of those trials are using restricted promoters from human genes. Promoters that restrict expression have demonstrat...

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Detalles Bibliográficos
Autores principales:	Simpson, Elizabeth M., Korecki, Andrea J., Fornes, Oriol, McGill, Trevor J., Cueva-Vargas, Jorge Luis, Agostinone, Jessica, Farkas, Rachelle A., Hickmott, Jack W., Lam, Siu Ling, Mathelier, Anthony, Renner, Lauren M., Stoddard, Jonathan, Zhou, Michelle, Di Polo, Adriana, Neuringer, Martha, Wasserman, Wyeth W.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Mary Ann Liebert, Inc., publishers 2019
Materias:	Research Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6437624/ https://www.ncbi.nlm.nih.gov/pubmed/30062914 http://dx.doi.org/10.1089/hum.2018.118

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