第13巻 2017年4月　第2号

■綜説

眼科分野における遺伝子導入法の開発
五十嵐　勉^1,2, 三宅　弘一², 小林　舞香^1,2, 高橋　和久^1,2, 中元　兼二¹, 岡田　尚巳², 高橋　浩^1
1日本医科大学眼科
²日本医科大学生化学・分子生物学（分子遺伝学）

New innovations for ocular gene therapy
Tsutomu Igarashi^1,2, Koichi Miyake², Maika Kobayashi^1,2, Kazuhisa Takahashi^1,2, Kenji Nakamoto¹, Takashi Okada² and Hiroshi Takahashi^1
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

Key words
gene therapy, adeno-associated virus (AAV) vector, glaucoma, retina, intravitreal injection

Correspondence to
Tsutomu Igarashi, Department of Ophthalmology, Graduate School of Medicine, Nippon Medical School, 1-1-5 Sendagi, Bunkyo-ku, Tokyo 113-8602, Japan
E-mail：tutomu@nms.ac.jp

受付：2017年2月9日　受理：2017年3月7日