Clinical applications of retinal gene therapies

Xin Fu; Viet Anh Nguyen Huu; Yaou Duan; Daniel S Kermany; Carolina C S Valentim; Runze Zhang; Jie Zhu; Charlotte L Zhang; Xiaodong Sun; Kang Zhang

doi:10.1093/pcmedi/pby004

Clinical applications of retinal gene therapies

Precis Clin Med. 2018 Jun;1(1):5-20. doi: 10.1093/pcmedi/pby004. Epub 2018 Jun 1.

Authors

Xin Fu^{1

2}, Viet Anh Nguyen Huu^{1

2}, Yaou Duan², Daniel S Kermany^{1

2}, Carolina C S Valentim², Runze Zhang², Jie Zhu², Charlotte L Zhang², Xiaodong Sun³, Kang Zhang^{1

2

4}

Affiliations

¹ Guangzhou Women and Children's Medical Center, Guangzhou Medical University, Guangzhou, China.
² Shiley Eye Institute, Institute for Engineering in Medicine, Institute for Genomic Medicine, University of California, San Diego, La Jolla, California, USA.
³ Shanghai Key Laboratory of Ocular Fundus Diseases, Shanghai General Hospital, Shanghai Jiaodong University, Shanghai, China.
⁴ Molecular Medicine Research Center, West China Hospital, Sichuan University, Chengdu, China.

Abstract

Retinal degenerative diseases are a major cause of blindness. Retinal gene therapy is a trail-blazer in the human gene therapy field, leading to the first FDA approved gene therapy product for a human genetic disease. The application of Clustered Regularly Interspaced Short Palindromic Repeat/Cas9 (CRISPR/Cas9)-mediated gene editing technology is transforming the delivery of gene therapy. We review the history, present, and future prospects of retinal gene therapy.

Publication types

Review