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Ocular Gene Therapy—The Future Is Now

Ocular Gene Therapy—The Future Is Now Downloaded from http://journals.lww.com/apjoo by BhDMf5ePHKbH4TTImqenVA5KvPVPZ0P5BEgU+IUTEfzO/GUWifn2IfwcEVVH9SSn on 06/01/2020 EDITORIAL Suber S. Huang, Katherine High, and Raffaella Toso he confluence of physics, chemistry, and the emerging field of biology that resulted in Watson and T Crick’s identification of the double helix presaged the discovery of nucleosomes, endonucleases, gene amplification, complete sequencing of many genomes including our own, the creation of artificial chromosomes, and techniques for gene therapy. Today, we again step forward at the dawn of a new era of ocular therapeutics with the first successful phase 3 ocular gene therapy study (Spark Therapeutics RPE65 trial, NCT00999609). The simple concept of replacing a mutated disease-causing gene with a wild type copy found an ideal application in the treatment of inherited retinal dystrophies (IRD). The eye is easily accessible, im- munologically isolated by the blood-retina barrier, and has extraordinarily well-characterized anatomy, physiology, and genetics garnered over decades of discovery and meticulous clinical trials. The develop- ment of gene therapy for retinal disease started more than 20 years ago with the first adenovirus- mediated gene transfer into the retina of adult mice. In 2007, progressive refinement and innovation culminated in the first clinical trials for Leber congenital amaurosis (LCA), a rare inherited retinal http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png The Asia-Pacific Journal of Ophthalmology Wolters Kluwer Health

Ocular Gene Therapy—The Future Is Now

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Publisher
Wolters Kluwer Health
Copyright
Copyright © 2016 by Asia Pacific Academy of Ophthalmology
ISSN
2162-0989
eISSN
2475-5028
DOI
10.1097/APO.0000000000000216
pmid
27488063
Publisher site
See Article on Publisher Site

Abstract

Downloaded from http://journals.lww.com/apjoo by BhDMf5ePHKbH4TTImqenVA5KvPVPZ0P5BEgU+IUTEfzO/GUWifn2IfwcEVVH9SSn on 06/01/2020 EDITORIAL Suber S. Huang, Katherine High, and Raffaella Toso he confluence of physics, chemistry, and the emerging field of biology that resulted in Watson and T Crick’s identification of the double helix presaged the discovery of nucleosomes, endonucleases, gene amplification, complete sequencing of many genomes including our own, the creation of artificial chromosomes, and techniques for gene therapy. Today, we again step forward at the dawn of a new era of ocular therapeutics with the first successful phase 3 ocular gene therapy study (Spark Therapeutics RPE65 trial, NCT00999609). The simple concept of replacing a mutated disease-causing gene with a wild type copy found an ideal application in the treatment of inherited retinal dystrophies (IRD). The eye is easily accessible, im- munologically isolated by the blood-retina barrier, and has extraordinarily well-characterized anatomy, physiology, and genetics garnered over decades of discovery and meticulous clinical trials. The develop- ment of gene therapy for retinal disease started more than 20 years ago with the first adenovirus- mediated gene transfer into the retina of adult mice. In 2007, progressive refinement and innovation culminated in the first clinical trials for Leber congenital amaurosis (LCA), a rare inherited retinal

Journal

The Asia-Pacific Journal of Ophthalmology – Wolters Kluwer Health

Published: Aug 1, 2016

References

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