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

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

Abstract 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...

Full description

Bibliographic Details
Main Authors: Ivana Trapani, Pasqualina Colella, Andrea Sommella, Carolina Iodice, Giulia Cesi, Sonia de Simone, Elena Marrocco, Settimio Rossi, Massimo Giunti, Arpad Palfi, Gwyneth J Farrar, Roman Polishchuk, Alberto Auricchio
Format: Article
Language:English
Published: Springer Nature 2013-12-01
Series:EMBO Molecular Medicine
Subjects:
AAV
ABCA4
gene therapy
MYO7A
retina
Online Access:https://doi.org/10.1002/emmm.201302948

Internet

https://doi.org/10.1002/emmm.201302948

Similar Items