HUNTINGTON’S DISEASE

A small molecule kicks repeat expansion into reverse

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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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 (2020). https://doi.org/10.1038/s41588-020-0577-6

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