A small molecule kicks repeat expansion into reverse

Michael Flower

doi:10.1038/s41588-020-0577-6

2020-02-14

PRE

Michael Flower

Sarah J. Tabrizi *

DOI：10.1038/s41588-020-0577-6

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摘要

En 中文

Huntington's disease is a severe progressive neurological disorder caused by a CAG-repeat expansion in the HTT gene. A small molecule shows therapeutic potential by inducing contraction of these expanded CAG repeats in cell and mouse models of the disease.

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Nature Reviews Endocrinology

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1.0W

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University of London

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