Let There Be Light: Gene and Cell Therapy for Blindness.

Abstract

Retinal degenerative diseases are a leading cause of irreversible blindness. Retinal cell death is the main cause of vision loss in genetic disorders such as retinitis pigmentosa, Stargardt disease, and Leber congenital amaurosis, as well as in complex age-related diseases such as age-related macular degeneration. For these blinding conditions, gene and cell therapy approaches offer therapeutic intervention at various disease stages. The present review outlines advances in therapies for retinal degenerative disease, focusing on the progress and challenges in the development and clinical translation of gene and cell therapies. A significant body of preclinical evidence and initial clinical results pave the way for further development of these cutting edge treatments for patients with retinal degenerative disorders.

• Authors: Deniz Dalkara, Olivier Goureau, Katia Marazova, José-Alain Sahel
• Journal: Human gene therapy (Hum Gene Ther) Vol. 27 Issue 2 Pg. 134-47 (Feb 2016) ISSN: 1557-7422 [Electronic] United States
• PMID: 26751519 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't, Review)

Chemical References

• retinoid isomerohydrolase
• cis-trans-Isomerases

Topics

• Adenoviruses, Human (genetics)
• Blindness (genetics, metabolism, pathology, therapy)
• CRISPR-Cas Systems
• Cell- and Tissue-Based Therapy (methods)
• Clinical Trials as Topic
• Dependovirus (genetics)
• Genetic Therapy (methods)
• Genetic Vectors (chemistry, metabolism)
• Humans
• Leber Congenital Amaurosis (genetics, metabolism, pathology, therapy)
• Lentivirus (genetics)
• Macular Degeneration (congenital, genetics, metabolism, pathology, therapy)
• Optogenetics (methods)
• Retina (metabolism, pathology)
• Retinitis Pigmentosa (genetics, metabolism, pathology, therapy)
• Stargardt Disease
• cis-trans-Isomerases (genetics, metabolism)