Attenuation of Inherited and Acquired Retinal Degeneration Progression with Gene-based Techniques.

Authors

Galaxy Y Cho, Jonas Children's Vision Care and Bernard & Shirlee Brown Glaucoma Laboratory; Columbia University; Quinnipiac University
Kyle Bolo, Columbia University
Karen Sophia Park, Jonas Children's Vision Care and Bernard & Shirlee Brown Glaucoma Laboratory; Columbia University
Jesse D Sengillo, Reading Hospital-Tower Health
Stephen H Tsang, Jonas Children's Vision Care and Bernard & Shirlee Brown Glaucoma Laboratory; Columbia UniversityFollow

Document Type

Article

Publication Date

2-23-2019

Abstract

Inherited retinal dystrophies cause progressive vision loss and are major contributors to blindness worldwide. Advances in gene therapy have brought molecular approaches into the realm of clinical trials for these incurable illnesses. Select phase I, II and III trials are complete and provide some promise in terms of functional outcomes and safety, although questions do remain over the durability of their effects and the prevalence of inflammatory reactions. This article reviews gene therapy as it can be applied to inherited retinal dystrophies, provides an update of results from recent clinical trials, and discusses the future prospects of gene therapy and genome surgery.

Publication Title

Mol Diagn Ther

Volume

23

Issue

1

First Page

113

Last Page

120

Recommended Citation

Cho, G., Bolo, K., Park, K., Sengillo, J., & Tsang, S. (2019). Attenuation of Inherited and Acquired Retinal Degeneration Progression with Gene-based Techniques.. Mol Diagn Ther, 23 (1), 113-120. https://doi.org/10.1007/s40291-018-0377-1