1. Molecular Therapies for Inherited Retinal Diseases-Current Standing, Opportunities and Challenges.
- Author
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Vázquez-Domínguez I, Garanto A, and Collin RWJ
- Subjects
- Clinical Trials as Topic, Gene Transfer Techniques adverse effects, Genetic Therapy adverse effects, Humans, Neuroprotective Agents therapeutic use, Retinal Degeneration drug therapy, Retinal Degeneration genetics, Genetic Therapy methods, Retinal Degeneration therapy
- Abstract
Inherited retinal diseases (IRDs) are both genetically and clinically highly heterogeneous and have long been considered incurable. Following the successful development of a gene augmentation therapy for biallelic RPE65 -associated IRD, this view has changed. As a result, many different therapeutic approaches are currently being developed, in particular a large variety of molecular therapies. These are depending on the severity of the retinal degeneration, knowledge of the pathophysiological mechanism underlying each subtype of IRD, and the therapeutic target molecule. DNA therapies include approaches such as gene augmentation therapy, genome editing and optogenetics. For some genetic subtypes of IRD, RNA therapies and compound therapies have also shown considerable therapeutic potential. In this review, we summarize the current state-of-the-art of various therapeutic approaches, including the pros and cons of each strategy, and outline the future challenges that lie ahead in the combat against IRDs.
- Published
- 2019
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