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Co-opting endogenous microRNAs for therapy

Nature Medicine volume 18, pages 1011–1012 (2012)Cite this article

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A recent study highlights the potential of therapeutically modulating the endogenous miRNA pathway in a mouse model of spinal and bulbar muscular atrophy (SBMA). The overexpression of a naturally occurring miRNA led to the downregulation of the mutant androgen receptor transcript as well as the polyglutamine-containing protein it encodes, both of which may contribute to pathogenesis in SBMA (pages 1136–1141).

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Figure 1: An miR-196a–mediated therapy of SBMA.

Katie Vicari

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  1. and the Department of Molecular Genetics, Christopher E. Pearson is in the Program of Genetics and Genome Biology, The Hospital for Sick Children, Toronto, Ontario, Canada, University of Toronto, Toronto, Ontario, Canada.,

    Christopher E Pearson

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  1. Christopher E Pearson
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Correspondence to Christopher E Pearson.

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Pearson, C. Co-opting endogenous microRNAs for therapy. Nat Med 18, 1011–1012 (2012). https://doi.org/10.1038/nm.2864

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