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Huntington's disease (HD)

Huntingtin (HTT); microRNA-196a (miR-196a)

Mouse and cell culture studies suggest miR-196a could help treat HD. In a mouse model for HD, miR-196a overexpression decreased HD-associated motor deficits and tissue pathology in the brain compared with normal miR-196a expression. In induced pluripotent stem (iPS) cells derived from a patient with HD and differentiated into neuronal cells, miR-196a decreased the accumulation of mutant HTT compared with no miR-196a. Next steps include developing a methodology for in vivo delivery of miR-196a.

SciBX 6(30); doi:10.1038/scibx.2013.798
Published online Aug. 8, 2013

Unpatented; licensing status not applicable

Cheng, P.-H. et al. Am. J. Hum. Genet.; published online June 27, 2013;
Contact: Shang-Hsun Yang, National Cheng Kung University, Tainan, Taiwan