Modified Viral Vectors Could Make Gene Therapies Safer in the Nervous System (2 of 4) (IMAGE)

American Association for the Advancement of Science (AAAS)

Modified Viral Vectors Could Make Gene Therapies Safer in the Nervous System (2 of 4)

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Delivering adeno-associated virus (AAV) vectors into the central nervous system (CNS) of nonhuman primates is associated with toxicity of sensory neurons in the dorsal root ganglia (DRG), which conventional immune suppression regimens do not prevent. Hordeaux et al. exploited a specific microRNA (miR183) that is largely restricted to DRG neurons, by including specific miR183 targets in the AAV vectors that reduced toxicity in DRG neurons. Their strategy could inform safer AAV-based gene therapies for many central nervous system diseases. This material relates to a paper that appeared in the Nov. 11, 2020, issue of Science Translational Medicine, published by AAAS. The paper, by J. Hordeaux at University of Pennsylvania in Philadelphia, PA; and colleagues was titled, "MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates."

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[Credit: Gene Therapy Program, University of Pennsylvania]

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