Gene therapy for blindness

Sight-restoring therapy for the visually impaired and blind is a major unmet medical need. Ocular gene therapy is a rational choice for restoring vision or preventing the loss of vision because most blinding diseases originate in cellular components of the eye, a compartment that is optimally suited...

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Bibliographic Details
Published in:Annual review of neuroscience 2013-07, Vol.36 (1), p.467-488
Main Authors: Sahel, José-Alain, Roska, Botond
Format: Article
Language:English
Subjects:
Animals
Blindness
Blindness - genetics
Blindness - therapy
Brain
Eyes & eyesight
Gene therapy
Genetic Therapy - methods
Genetics
Humans
Neurobiology
Recovery of Function - genetics
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Summary:Sight-restoring therapy for the visually impaired and blind is a major unmet medical need. Ocular gene therapy is a rational choice for restoring vision or preventing the loss of vision because most blinding diseases originate in cellular components of the eye, a compartment that is optimally suited for the delivery of genes, and many of these diseases have a genetic origin or genetic component. In recent years we have witnessed major advances in the field of ocular gene therapy, and proof-of-concept studies are under way to evaluate the safety and efficacy of human gene therapies. Here we discuss the concepts and recent advances in gene therapy in the retina. Our review discusses traditional approaches such as gene replacement and neuroprotection and also new avenues such as optogenetic therapies. We conjecture that advances in gene therapy in the retina will pave the way for gene therapies in other parts of the brain.
ISSN:0147-006X
1545-4126
DOI:10.1146/annurev-neuro-062012-170304