Ontology highlight
ABSTRACT:
SUBMITTER: Manfredi A
PROVIDER: S-EPMC3868409 | biostudies-literature | 2013 Dec
REPOSITORIES: biostudies-literature
Manfredi Anna A Marrocco Elena E Puppo Agostina A Cesi Giulia G Sommella Andrea A Della Corte Michele M Rossi Settimio S Giunti Massimo M Craft Cheryl M CM Bacci Maria Laura ML Simonelli Francesca F Surace Enrico M EM Auricchio Alberto A
Human gene therapy 20131030 12
Gene transfer to both cone and rod photoreceptors (PRs) is essential for gene therapy of inherited retinal degenerations that are caused by mutations in genes expressed in both PR types. Vectors based on the adeno-associated virus (AAV) efficiently transduce PRs of different species. However, these are predominantly rods and little is known about the ability of the AAV to transduce cones in combination with rods. Here we show that AAV2/8 transduces pig cones to levels that are similar to AAV2/9, ...[more]