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