Gene therapy for retinal disease.

Michelle E McClements, Robert E MacLaren

Translational research : the journal of laboratory and clinical medicine 2013 Apr

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Gene therapy strategies for the treatment of inherited retinal diseases have made major advances in recent years. This review focuses on adeno-associated viral (AAV) vector approaches to treat retinal degeneration and, thus, prevent or delay the onset of blindness. Data from human clinical trials of gene therapy for retinal disease show encouraging signs of safety and efficacy from AAV vectors. Recent progress in enhancing cell-specific targeting and transduction efficiency of the various retinal layers plus the use of AAV-delivered growth factors to augment the therapeutic effect and limit cell death suggest even greater success in future human trials is possible. Copyright © 2013 Mosby, Inc. All rights reserved.

Citation

Michelle E McClements, Robert E MacLaren. Gene therapy for retinal disease. Translational research : the journal of laboratory and clinical medicine. 2013 Apr;161(4):241-54