Safety of Intravitreal Gene Therapy for Treatment of Subjects with Leber Hereditary Optic Neuropathy due to Mutations in the Mitochondrial ND4 Gene: The REVEAL Study

BACKGROUND: Leber hereditary optic neuropathy (LHON) is a maternally inherited mitochondrial disease whose primary clinical manifestation is bilateral visual loss. Only a single therapy, idebenone, is approved in Europe for use in exceptional circumstances and no therapy is currently approved in the...

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Detalles Bibliográficos
Autores principales: Vignal-Clermont, Catherine, Girmens, Jean-François, Audo, Isabelle, Said, Saddek Mohand, Errera, Marie-Hélène, Plaine, Lise, O’Shaughnessy, Denis, Taiel, Magali, Sahel, José-Alain
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2021
Materias:
Original Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7952288/
https://www.ncbi.nlm.nih.gov/pubmed/33566264
http://dx.doi.org/10.1007/s40259-021-00468-9

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7952288/
https://www.ncbi.nlm.nih.gov/pubmed/33566264
http://dx.doi.org/10.1007/s40259-021-00468-9

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