Clinical applications of retinal gene therapies.
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ABSTRACT:
SUBMITTER: Fu X
PROVIDER: S-EPMC8982485 | biostudies-literature | 2018 Jun
REPOSITORIES: biostudies-literature
Publications
Clinical applications of retinal gene therapies.
Fu Xin X Huu Viet Anh Nguyen VAN Duan Yaou Y Kermany Daniel S DS Valentim Carolina C S CCS Zhang Runze R Zhu Jie J Zhang Charlotte L CL Sun Xiaodong X Zhang Kang K
Precision clinical medicine 20180601 1
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. ...[more]
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