Adeno-associated viral vectors for retinal gene transfer.
Vectors derived from adeno-associated viruses (AAV) represent a promising tool for retinal gene transfer in pre-clinical and clinical settings. AAV vectors efficiently transduce dividing and non-dividing cells, escape cellular immunity and result in long-non-term transduction. In addition, they may be targeted to specific retinal cell types by taking advantage of surface proteins from various AAV serotypes thus limiting transfer of therapeutic genes to those cells requiring correction. This review will provide an overview of the properties of AAV vectors followed by a detailed report of their use in retinal gene transfer for mendelian and non-mendelian disorders.
Telethon Institute of Genetics and Medicine, Via P. Castellino 111, Naples 80131, Italy.
Enrico M
EM
Surace
Alberto
A
Auricchio
D000818:Animals; D000229:Dependovirus / Q000235:genetics*; D018390:Gene Targeting; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D012160:Retina; D012164:Retinal Diseases / Q000628:therapy; D013812:Therapeutics / Q000379:methods