Gene therapy beyond luxturna: a new horizon of the treatment for inherited retinal disease.

Journal Article (Journal Article;Review)

PURPOSE OF REVIEW: Gene therapy offers, for the first time, the possibility to cure diseases such as retinitis pigmentosa. The positive outcomes that led to the U.S. Food and Drug Administration (FDA) approval of Luxturna to treat Leber congenital amaurosis caused by RPE65 mutations created an optimistic atmosphere in the research, clinical and patient community. Despite this first success, we must understand that this is not a 'one treatment for all'. This review aims to explain the basic concepts of gene therapy and how they translate in different approaches that are utilized in ongoing clinical trials here reviewed. RECENT FINDINGS: In 2017, the FDA approved the first gene therapy treatment. In parallel, other approaches have gained attention. Different delivery methods (adeno-associated virus, lentivirus), injection sites (subretinal, intravitreal, suprachoroidal) and methodologies (gene replacement, silencing, editing) are currently being tested. SUMMARY: Gene therapy is an evolving field in medicine and ophthalmology. Its success and application depends on several factors that are specific to the disease to treat. For now, we know it's a relatively safe approach and we look forward to the continued advancements of current ongoing clinical trials.

Full Text

Duke Authors

Cited Authors

  • Prado, DA; Acosta-Acero, M; Maldonado, RS

Published Date

  • May 2020

Published In

Volume / Issue

  • 31 / 3

Start / End Page

  • 147 - 154

PubMed ID

  • 32175942

Electronic International Standard Serial Number (EISSN)

  • 1531-7021

Digital Object Identifier (DOI)

  • 10.1097/ICU.0000000000000660


  • eng

Conference Location

  • United States