title: [The development in the studies of AAV-mediated gene therapy in retina].
abstract: Significant progress in understanding the molecular basis of retinal disorders has led to the development of gene therapies for treatment of these diseases. Adeno-associated virus (AAV) is a useful vector for the treatment of retinal diseases due to its low toxicity and immunogenicity, ability to transducer both dividing and non-dividing cells, and stable transgene expression. A variety of animal studies and clinical trials have proved the safety and effectivity of retinal AAV-mediated gene therapy. AAV-mediated gene therapy, such as anti-angiogenic proteins, neurotrophic factors, anti-apoptosis factors were studied in animal disease models, and the results were satisfactory. However, the main drawback of AAV vectors is its relatively small packaging capacity, which needs further improvement.
authors.affiliation: Department of Ophthalmology, Peking Union Medical College Hospital, Peking Union Medical College, Chinese Academy of Medical Sciences, Beijing 100730, China.
authors.forename: Xuan
authors.initials: X
authors.lastname: Zou
authors.forename: Rui-fang
authors.initials: RF
authors.lastname: Sui
authors.forename: Fang-tian
authors.initials: FT
authors.lastname: Dong
mesh_terms: D000818:Animals; D000229:Dependovirus / Q000235:genetics*; D004195:Disease Models, Animal; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006801:Humans; D012164:Retinal Diseases / Q000628:therapy*