■特集〔遺伝子治療update：日本医科大学の遺伝子治療研究（9）〕
眼科分野における遺伝子治療
五十嵐 勉
日本医科大学千葉北総病院眼科
日本医科大学生化学・分子生物学（分子遺伝学）
Gene Therapy for Ophthalmic Diseases
Tsutomu Igarashi
Department of Ophthalmology, Nippon Medical School Chiba Hokusoh Hospital
Department of Biochemistry and Molecular Biology, Nippon Medical School
In the field of ophthalmology, gene therapy has focused on hereditary retinal dystrophy, including retinitis pigmentosa. Hereditary retinal dystrophy is a group of intractable diseases for which there is currently no effective treatment, and expectations are focused on gene therapy and retinal regeneration therapy using iPS cells. In recent years, results of a number of clinical trials of gene therapies for hereditary retinal dystrophy have been reported from Europe and the United States. Since 2017, gene therapy drugs for Leber congenital amaurosis have been approved after being found to be safe and therapeutically efficacious. In this article, we will discuss the current state of gene therapy for retinitis pigmentosa and the use of adeno-associated virus (AAV) vectors in gene therapy.
日医大医会誌 2023; 19(3), 242-246
Key words
gene therapy, ophthalmology, retina, retinitis pigmentosa, hereditary retinal disease
受付：2023年3月27日 受理：2023年7月3日 |
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