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Lab Reports |

Scientists Apply Gene Therapy to Treat Blinding Eye Diseases in Animals

Tracy Hampton, PhD
JAMA. 2013;310(5):470. doi:10.1001/jama.2013.110987.
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A new gene therapy vector provides a noninvasive way to penetrate deep into the eye’s outer retina and it appears to effectively treat 2 different retinal diseases in mice, report researchers from the University of California, Berkeley (Dalkara D et al. Sci Transl Med. 2013;5[189]:189ra76). The vector can also transfer genes deep into the healthy retinas of monkeys, reaching rod and cone photoreceptor cells by traveling through the vitreous cavity.

The technique appears to be safer and more effective than current gene delivery methods, which require a subretinal injection and transduce just a fraction of the cells in the retina.

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A new gene therapy vector can deliver a gene—in this case, one encoding a green fluorescent protein—to all retinal layers in a nonhuman primate without the need for invasive injections.

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