Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus

Usher 1 patients are born profoundly deaf and then develop retinal degeneration. Thus they are readily identified prior to the onset of retinal degeneration, making gene therapy a viable strategy to prevent their blindness. Here, we have investigated the use of adeno-associated viruses (AAV) for the...

Descripción completa

Detalles Bibliográficos
Autores principales: Lopes, Vanda S., Boye, Shannon E., Louie, Carrie M., Boye, Sanford, Dyka, Frank, Chiodo, Vince, Fofo, Hugo, Hauswirth, William W., Williams, David S.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2013
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3640772/
https://www.ncbi.nlm.nih.gov/pubmed/23344065
http://dx.doi.org/10.1038/gt.2013.3

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3640772/
https://www.ncbi.nlm.nih.gov/pubmed/23344065
http://dx.doi.org/10.1038/gt.2013.3

Ejemplares similares