Effective delivery of large genes to the retina by dual AAV vectors

Retinal gene therapy with adeno-associated viral (AAV) vectors is safe and effective in humans. However, AAV's limited cargo capacity prevents its application to therapies of inherited retinal diseases due to mutations of genes over 5 kb, like Stargardt's disease (STGD) and Usher syndrome...

Descripción completa

Detalles Bibliográficos
Autores principales: Trapani, Ivana, Colella, Pasqualina, Sommella, Andrea, Iodice, Carolina, Cesi, Giulia, de Simone, Sonia, Marrocco, Elena, Rossi, Settimio, Giunti, Massimo, Palfi, Arpad, Farrar, Gwyneth J, Polishchuk, Roman, Auricchio, Alberto
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Blackwell Publishing Ltd 2014
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3927955/
https://www.ncbi.nlm.nih.gov/pubmed/24150896
http://dx.doi.org/10.1002/emmm.201302948

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3927955/
https://www.ncbi.nlm.nih.gov/pubmed/24150896
http://dx.doi.org/10.1002/emmm.201302948

Ejemplares similares