Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement

Leber congenital amaurosis (LCA) associated with retinal pigment epithelium-specific protein 65 kDa (RPE65) mutations is a severe hereditary blindness resulting from both dysfunction and degeneration of photoreceptors. Clinical trials with gene augmentation therapy have shown partial reversal of the...

Descripción completa

Guardado en:
Detalles Bibliográficos
Autores principales: Cideciyan, Artur V., Jacobson, Samuel G., Beltran, William A., Sumaroka, Alexander, Swider, Malgorzata, Iwabe, Simone, Roman, Alejandro J., Olivares, Melani B., Schwartz, Sharon B., Komáromy, András M., Hauswirth, William W., Aguirre, Gustavo D.
Formato: Artigo
Lenguaje:Inglês
Publicado: National Academy of Sciences 2013
Materias:
PNAS Plus
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC3568385/
https://ncbi.nlm.nih.gov/pubmed/23341635
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1218933110
Etiquetas: Agregar Etiqueta
Sin Etiquetas, Sea el primero en etiquetar este registro!

Ejemplares similares