Randomized trial of bilateral gene therapy injection for m.11778G>A MT-ND4 Leber optic neuropathy

Leber hereditary optic neuropathy (LHON) is an important example of mitochondrial blindness with the m.11778G>A mutation in the MT-ND4 gene being the most common disease-causing mtDNA variant worldwide. The REFLECT phase 3 pivotal study is a randomized, double-masked, placebo-controlled trial inv...

Descripción completa

Detalles Bibliográficos
Autores principales: Newman, Nancy J, Yu-Wai-Man, Patrick, Subramanian, Prem S, Moster, Mark L, Wang, An-Guor, Donahue, Sean P, Leroy, Bart P, Carelli, Valerio, Biousse, Valerie, Vignal-Clermont, Catherine, Sergott, Robert C, Sadun, Alfredo A, Rebolleda Fernández, Gema, Chwalisz, Bart K, Banik, Rudrani, Bazin, Fabienne, Roux, Michel, Cox, Eric D, Taiel, Magali, Sahel, José-Alain
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2022
Materias:
Clinical Trial
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10115230/
https://www.ncbi.nlm.nih.gov/pubmed/36350566
http://dx.doi.org/10.1093/brain/awac421

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10115230/
https://www.ncbi.nlm.nih.gov/pubmed/36350566
http://dx.doi.org/10.1093/brain/awac421

Ejemplares similares