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Recent Advances in RNA Interference Therapeutics for CNS Diseases

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Neurotherapeutics

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.

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Acknowledgments

We thank members of the Davidson Lab for critical comments, and the NIH, NAF, HDF, and Roy J Carver Trust for funding this work.

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Authors and Affiliations

  1. Interdisciplinary program in Genetics, University of Iowa, Iowa City, IA, 52242, USA

    Pavitra S. Ramachandran & Beverly L. Davidson

  2. Interdisciplinary program in Neuroscience, University of Iowa, Iowa City, IA, USA

    Megan S. Keiser & Beverly L. Davidson

  3. Department of Internal Medicine, University of Iowa, Iowa City, USA

    Beverly L. Davidson

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  1. Pavitra S. Ramachandran
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  2. Megan S. Keiser
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  3. Beverly L. Davidson
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Correspondence to Beverly L. Davidson.

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P. S. Ramachandran and M. S. Keiser contributed equally to this work.

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Ramachandran, P.S., Keiser, M.S. & Davidson, B.L. Recent Advances in RNA Interference Therapeutics for CNS Diseases. Neurotherapeutics 10, 473–485 (2013). https://doi.org/10.1007/s13311-013-0183-8

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