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National Institute of Environmental Health Sciences

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Publication Detail

Title: Recent advances in RNA interference therapeutics for CNS diseases.

Authors: Ramachandran, Pavitra S; Keiser, Megan S; Davidson, Beverly L

Published In Neurotherapeutics, (2013 Jul)

Abstract: Over the last decade, RNA interference technology has shown therapeutic promise in rodent models of dominantly inherited brain diseases, including those caused by polyglutamine repeat expansions in the coding region of the affected gene. For some of these diseases, proof-of concept studies in model organisms have transitioned to safety testing in larger animal models, such as the nonhuman primate. Here, we review recent progress on RNA interference-based therapies in various model systems. We also highlight outstanding questions or concerns that have emerged as a result of an improved (and ever advancing) understanding of the technologies employed.

PubMed ID: 23589092 Exiting the NIEHS site

MeSH Terms: No MeSH terms associated with this publication

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