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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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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DOI: https://doi.org/10.1038/nm.2864