RESEARCH ARTICLE


Gene Therapy for Diabetic Retinopathy in Animal Models and Humans



Hiroaki Mizukami*, Masashi Urabe, Akihiro Kume, Keiya Ozawa
Division of Genetic Therapeutics, Center for Molecular Medicine, Jichi Medical University, 3311-1 Yakushiji, Shimotsuke, Tochigi, 329-0498, Japan.


© Mizukami et al.

open-access license: This is an open access article distributed under the terms of the Creative Commons Attribution 4.0 International Public License (CC-BY 4.0), a copy of which is available at: https://creativecommons.org/licenses/by/4.0/legalcode. This license permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.

* Address correspondence to this author at the Division of Genetic Therapeutics, Center for Molecular Medicine, Jichi Medical University, 3311-1 Yakushiji, Shimotsuke, Tochigi, 329-0498, Japan. Tel: +81-285-58- 7402; Fax: +81-285-44-8675; E-mail: miz@jichi.ac.jp


Abstract

Gene therapy is considered as one of the innovative treatment modalities for diabetic retinopathy (DR). Since genuine animal models of DR are limited, only a few studies have reported the efficacy of gene therapy. For preclinical study of DR, spontaneously diabetic Torii (SDT) rat is a valuable model. Fortunately, we could evaluate the efficacy of adeno-associated virus (AAV)-mediated gene therapy in SDT rats and proved that sFlt-1 expression prevented DR progression. Because of a limited number of large-animal models of DR, it is uncertain whether gene therapy experiments using dogs or monkeys allow reliable conclusions. On the other hand, owing to the recent progress in AAV-mediated gene therapy for retinal diseases in monkeys and humans, gene therapy for DR using AAV vectors may become a reality in the near future.

Keywords: Diabetic retinopathy, SDT rat, gene therapy, AAV vector.