Journal
TRANSLATIONAL RESEARCH
Volume 161, Issue 4, Pages 241-254Publisher
ELSEVIER SCIENCE INC
DOI: 10.1016/j.trsl.2012.12.007
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Funding
- National Institute for Health Research Biomedical Research Centres at Oxford University Hospitals NHS Trust
- Moorfields Eye Hospital
- UK Medical Research Council
- Wellcome Trust
- Health Foundation
- Royal College of Surgeons of Edinburgh
- Royal Society
- Fight for Sight
- Lanvern Foundation
- Oxford Stem Cell Institute
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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. (Translational Research 2013;161:241-254)
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