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Sub-foveal gene therapy treatment of 6 choroideremia patients show improved rod and cone photoreceptor cell function

Researchers based at the Nuffield Laboratory of Ophthalmology, Department of Clinical Neurosciences, University of Oxford, and at Imperial College London, UK, have published research findings suggesting that an experimental gene therapy treatment of 6 male patients with choroideremia shows rescue of rod and cone cell function. According to the authors of the study, the research, published in the Lancet (January 16, 2014; reports data from the first human gene therapy trial targeting genes expressed in photoreceptor cells. The X-linked recessive indication has a prevalence of approximately 1 in 50,000 and is currently incurable. Successful proof-of-concept holds significant implication not only for treatment of choroideremia but also for treatment of a number of other retinal degenerations including retinitis pigmentosa (RP) and age-related macular degeneration (AMD).