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HUNTINGTON’S DISEASE

A small molecule kicks repeat expansion into reverse

Nature Genetics volume 52, pages 136–137 (2020)Cite this article

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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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Fig. 1: NA induces contraction of CAG repeats in a mouse model of HD.

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Authors and Affiliations

  1. University College London (UCL) Huntington’s Disease Centre, Department of Neurodegenerative Disease, UCL Queen Square Institute of Neurology and Dementia Research Centre at UCL, London, UK

    Michael D. Flower & Sarah J. Tabrizi

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  1. Michael D. Flower
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  2. Sarah J. Tabrizi
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Correspondence to Sarah J. Tabrizi.

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Competing interests

M.D.F. has no competing interests. S.J.T. is on scientific advisory boards for Triplet Therapeutics and LoQus therapeutics and has laboratory funding from Vertex pharmaceuticals.

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Flower, M.D., Tabrizi, S.J. A small molecule kicks repeat expansion into reverse. Nat Genet 52, 136–137 (2020). https://doi.org/10.1038/s41588-020-0577-6

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