Ontology highlight
ABSTRACT:
SUBMITTER: Trapani I
PROVIDER: S-EPMC3927955 | biostudies-literature | 2014 Feb
REPOSITORIES: biostudies-literature
EMBO molecular medicine 20131215 2
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 type IB (USH1B). Previous methods based on 'forced' packaging of large genes into AAV capsids may not be easily translated to the clinic due to the generation of genomes of heterogeneous size which raise safety ...[more]