Richard Masoner / Cyclelicious [creativecommons license] via Wikimedia CommonsHereditary retinal degeneration, in which mutations in photoreceptors and other delicate cells of the eye’s outermost retinal layer lead to deterioration and visual impairment, affects roughly 1 in 3,000 people worldwide. Gene therapy can successfully deliver normal copies of the mutated genes to the affected cells using adeno-associated virus (AAV) but requires that the virus be injected through the retina. These injections reach only a fraction of the affected cells and, moreover, can damage the fragile retina, making them unsuitable for treating the many forms of retinal degeneration in which the retinal tissue is structurally compromised. A technique recently published in Science Translational Medicine (5, 189ra76; 2013) addresses the resultant need for a less invasive method of delivering gene therapy across the retina.
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