Gene therapy in inherited retinal degenerative diseases, a review.

Ophthalmic Genet

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

Published: October 2018

Hereditary diseases of the retina represent a group of diseases with several heterogeneous mutations that have the common end result of progressive photoreceptor death leading to blindness. Retinal degenerations encompass multifactorial diseases such as age-related macular degeneration, Leber congenital amaurosis, Stargardt disease, and retinitis pigmentosa. Although there is currently no cure for degenerative retinal diseases, ophthalmology has been at the forefront of the development of gene therapy, which offers hope for the treatment of these conditions. This article will explore an overview of the clinical trials of gene supplementation therapy for retinal diseases that are underway or planned for the near future.

Download full-text PDF

Source
http://dx.doi.org/10.1080/13816810.2018.1495745DOI Listing

Publication Analysis

Top Keywords

gene therapy
8
retinal diseases
8
diseases
6
therapy inherited
4
retinal
4
inherited retinal
4
retinal degenerative
4
degenerative diseases
4
diseases review
4
review hereditary
4

Similar Publications

Want AI Summaries of new PubMed Abstracts delivered to your In-box?

Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!