17 Jul Gene Therapy May Stop Blindness From Retinitis Pigmentosa
MedicalResearch.com Interview with:
Professor Robert E MacLaren MB ChB DPhil FRCOphth FRCS
Nuffield Laboratory of Ophthalmology
Nuffield Department of Clinical Neurosciences
Oxford Biomedical Research Centre, University of Oxford,
Moorfields Eye Hospital & UCL NIHR Biomedical Research Centre for Ophthalmology
Medical Research: What is the background for this study? What are the main findings?
Prof. MacLaren: The study shows that gene therapy can be used to release a protein in the eye that arrests the development of retinitis pigmentosa, a blinding disease caused by degeneration of the retina. The study was performed in mice which had a similar genetic defect to that found in humans with the disease. The mice also had fluorescent green “glow in the dark” light sensing cells known as cones, which we could see and count by looking into the eye – like counting stars in the night sky. By counting the green fluorescent cones we were able to work out the exact dose of gene therapy needed to keep these cells alive indefinitely. The study was funded by Fight for Sight, a UK charity that supports finding cures for eye diseases.
Medical Research: What should clinicians and patients take away from your report?
Prof. MacLaren: Gene therapy has great promise in treating retinitis pigmentosa but we are only just entering clinical trials and there is a lot more to do before it becomes an approved treatment.
Medical Research: What recommendations do you have for future research as a result of this study?
Prof. MacLaren: We need to know more about how the protein induced by the gene therapy works. The protein is known as ciliary neurotrophic factor (CNTF) and has been in clinical trials before but some toxic effects were seen. It may be more stable when delivered at a lower dose using gene therapy, or the route of administration may be critical to the success with retinitis pigmentosa. We now have sufficient data to start designing a clinical trial.
Mol Ther. 2015 Apr 21. doi: 10.1038/mt.2015.68. [Epub ahead of print]
CNTF Gene Therapy Confers Lifelong Neuroprotection in a Mouse Model of Human Retinitis Pigmentosa.
Lipinski DM1, Barnard AR2, Singh MS2, Martin C3, Lee EJ4, Davies WI5, MacLaren RE6.
Professor Robert E MacLaren MB ChB DPhil (2015). Gene Therapy May Stop Blindness From Retinitis Pigmentosa