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Failure of genetic therapies for Huntington’s devastates community

Hopes were high for drugs designed to lower levels of a mutant protein, but development has stalled.
  1. Diana Kwon
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Two pharmaceutical companies have halted clinical trials of gene-targeting therapies for Huntington’s disease (HD), following the drugs’ disappointing performance.

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Nature 593, 180 (2021)

doi: https://doi.org/10.1038/d41586-021-01177-7

Updates & Corrections

  • Clarification 06 May 2021: This article has been updated to include more detail about the ASOs developed by Wave Life Sciences.

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