New innovations for ocular gene therapy
1)Department of Ophthalmology, Nippon Medical School
2)Department of Biochemistry and Molecular Biology, Nippon Medical School
Adeno-associated virus (AAV) vectors are widely used for retinal gene transfer, and they are undergoing various clinical trials. Their popularity is due to the non-pathogenic nature of AAVs and their versatility in basic research and clinical applications; the excellent transduction efficiency of AAV vectors has boosted basic research and has facilitated the development of various technical innovation systems, such as AAV vector serotypes, self-complementary AAV vectors, tyrosine mutated AAV vectors and the routes of vector administration. However, while the transduction efficiency of intravitreal injections has increased markedly in rodents, it is still low in non-human primates. We have recently developed a new technique of intravitreal administration in macaque monkeys. In this review, we outline and discuss strategies for developing AAV vector systems and advancing intravitreal administration.
日医大医会誌 2017; 13(2), 88-96
gene therapy, adeno-associated virus (AAV) vector, glaucoma, retina, intravitreal injection
Tsutomu Igarashi, Department of Ophthalmology, Graduate School of Medicine, Nippon Medical School, 1-1-5 Sendagi, Bunkyo-ku, Tokyo 113-8602, Japan