RESEARCH ARTICLE
Gene Therapy for Diabetic Retinopathy in Animal Models and Humans
Hiroaki Mizukami*, Masashi Urabe, Akihiro Kume, Keiya Ozawa
Article Information
Identifiers and Pagination:
Year: 2011Volume: 4
First Page: 119
Last Page: 122
Publisher Id: TODIAJ-4-119
DOI: 10.2174/1876524601104010119
Article History:
Received Date: 27/10/2010Revision Received Date: 04/12/2010
Acceptance Date: 15/01/2011
Electronic publication date: 28/3/2011
Collection year: 2011
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.
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.