Pseudotyped AAV vectors for constitutive and regulated gene expression in the eye.
Since the first reports describing the injection of recombinant adeno-associated viral (AAV) vectors in the murine eye, the advancement of the field has been enormous resulting in the correction of several animal models of retinal diseases. The recent development of "pseudotyped" AAV vectors with transduction characteristics that best fit the correction of specific retinal disease phenotypes and of sophisticated systems for tight regulation of gene expression expands on the potentiality of this delivery system for the eye.
Auricchio
Alberto
A
Telethon Institute of Genetics and Medicine (TIGEM), Via P. Castellino 111, 80131, Napoli, Italy. auticchio@tigem.it
D000818:Animals; D000229:Dependovirus / Q000235:genetics*; D005786:Gene Expression Regulation; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000627:therapeutic use*; D006801:Humans; D051379:Mice; D012160:Retina / Q000378:metabolism; D012164:Retinal Diseases / Q000628:therapy*; D014161:Transduction, Genetic