Retinal gene therapy in X-linked retinitis pigmentosa caused by mutations in RPGR: Results at 6 months in a first in human clinical trial

Retinal gene therapy has shown great promise in treating retinitis pigmentosa (RP), a primary photoreceptor degeneration that leads to severe sight loss in young people(1,2,3,4,5,6). Here we report the first in human Phase I/II dose escalation clinical trial for X-linked RP caused by mutations in th...

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Detalles Bibliográficos
Autores principales: Cehajic-Kapetanovic, Jasmina, Xue, Kanmin, de la Camara, Cristina Martinez-Fernandez, Nanda, Anika, Davies, Alexandra, Wood, Laura J, Salvetti, Anna Paola, Fischer, M Dominik, Aylward, James W, Barnard, Alun R, Jolly, Jasleen K, Luo, Edmond, Lujan, Brandon J, Ong, Tuyen, Girach, Aniz, Black, Graeme CM, Gregori, Ninel Z, Davis, Janet L, Rosa, Potyra R, Lotery, Andrew J, Lam, Byron L, Stanga, Paulo E, MacLaren, Robert E
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7104347/
https://www.ncbi.nlm.nih.gov/pubmed/32094925
http://dx.doi.org/10.1038/s41591-020-0763-1

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7104347/
https://www.ncbi.nlm.nih.gov/pubmed/32094925
http://dx.doi.org/10.1038/s41591-020-0763-1

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