Gene Therapy Without Retinal Risk

Researchers have developed a method of delivering genes to the inner layers of the retina without the risks associated with injection into the delicate tissues themselves. This less invasive technique has been carried out successfully in animal models by a research team led by Drs. John G. Flannery and David V. Schaffer at the University of California, Berkeley. They narrowed their search to a variant of the traditional adeno-associated virus (AAV), which can more easily travel through the retinal layers after being injected into the vitreous gel of the eye. The vitreous is a much more attainable and less risky target.
The team is now planning to prepare what they call 7m8 AAV for study in humans, with hope of soon entering into clinical trials.
Abstract: “In vivo-directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous” by Dalkara D, Byrne LC, et al (Helen Wills Neuroscience Institute, University of California, Berkeley, CA 94720-1462, June 12, 2013)
Source: Sci Transl Med. 2013 Jun 12;5(189):189ra76. doi: 10.1126/scitranslmed.3005708

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