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Safety and improved efficacy signals following gene therapy in childhood blindness caused by GUCY2D mutations

A first-in-human clinical trial of gene therapy in Leber congenital amaurosis due to mutations in the GUCY2D gene is underway, and early results are summarized. A recombinant adeno-associated virus serotype 5 (rAAV5) vector carrying the human GUCY2D gene was delivered by subretinal injection to one...

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Detalles Bibliográficos
Autores principales:	Jacobson, Samuel G., Cideciyan, Artur V., Ho, Allen C., Peshenko, Igor V., Garafalo, Alexandra V., Roman, Alejandro J., Sumaroka, Alexander, Wu, Vivian, Krishnan, Arun K., Sheplock, Rebecca, Boye, Sanford L., Dizhoor, Alexander M., Boye, Shannon E.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier 2021
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8099775/ https://www.ncbi.nlm.nih.gov/pubmed/33997691 http://dx.doi.org/10.1016/j.isci.2021.102409

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