The research status of preclinical animal experiments of gene therapy for Leber congenital amaurosis

Abstract

Leber congenital amaurosis （LCA） is extremely severe hereditary retinal dystrophy, because it leads to congenital bilateral blindness in early childhood. With the development of molecular genetics and the therapeutic gene replacement technology, gene therapy clinical trials have obtained exciting results on the basis of relatively satisfied with preliminary clinical experimental results by adeno-associated virus （AAV） vector-mediated gene therapy in the past decade. These researching methods include intravitreal injection and subretinal space injection of gene vector, and the investigating indexes include evaluations of visual function and safety, such as immune reaction of the animals,ocular histopathological change,complications and bio-distribution of gene sequence. The preliminary success of the LCA17 ]I gene therapy will give some clues to the other inherited retinal diseases. This review focuses on the present status of pre-clinical animal experiments of its gene therapy. Key words: Leber congenital amaurosis; Gene therapy; RPE65 gene; Visual function; Safety