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

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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.

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Acknowledgements

The Jonas Children’s Vision Care and Bernard & Shirlee Brown Glaucoma Laboratory are supported by the National Institutes of Health (P30EY019007, R01EY018213, R01EY024698, R01EY026682, R21AG050437), National Cancer Institute Core (5P30CA013696), Foundation Fighting Blindness [TA-NMT-0116-0692-COLU], the Research to Prevent Blindness (RPB) Physician-Scientist Award, and unrestricted funds from RPB, New York, NY, USA. SHT is a member of the RD-CURE Consortium and is supported by Kobi and Nancy Karp, the Crowley Family Fund, the Rosenbaum Family Foundation, the Tistou and Charlotte Kerstan Foundation, the Schneeweiss Stem Cell Fund, New York State [C029572], and the Gebroe Family Foundation.

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  1. Galaxy Y. Cho and Kyle Bolo contributed equally to this paper.

Authors and Affiliations

  1. Jonas Children’s Vision Care and Bernard & Shirlee Brown Glaucoma Laboratory, New York, USA

    Galaxy Y. Cho, Karen Sophia Park & Stephen H. Tsang

  2. Department of Ophthalmology, Columbia University, New York, NY, USA

    Galaxy Y. Cho, Karen Sophia Park & Stephen H. Tsang

  3. Frank H. Netter MD School of Medicine, Quinnipiac University, North Haven, CT, USA

    Galaxy Y. Cho

  4. Vagelos College of Physicians and Surgeons, Columbia University, New York, NY, USA

    Kyle Bolo & Stephen H. Tsang

  5. Department of Internal Medicine, Reading Hospital, West Reading, PA, USA

    Jesse D. Sengillo

  6. Department of Pathology & Cell Biology, Stem Cell Initiative (CSCI), Institute of Human Nutrition, New York, NY, USA

    Stephen H. Tsang

  7. Harkness Eye Institute, Columbia University Medical Center, 635 West 165th Street, Box 212, New York, NY, 10032, USA

    Stephen H. Tsang

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GYC and KB performed the literature searches and composed the manuscript. KSP and JDS assisted in manuscript composition. SHT oversaw all aspects of the manuscript preparation and holds final responsibility for content and the decision to publish.

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Correspondence to Stephen H. Tsang.

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The authors Galaxy Y. Cho, Kyle Bolo, Karen Sophia Park, Jesse D. Sengillo, Stephen H. Tsang have no conflicts to declare.

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

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