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Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium
Published on: August 7, 2015
Clinical applications of retinal gene therapies
Xin Fu1,2, Viet Anh Nguyen Huu1,2, Yaou Duan2
1Guangzhou Women and Children's Medical Center, Guangzhou Medical University, Guangzhou, 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.
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