HDYO Research Update: Two Birds One Stone - HTT-lowering drugs also target CAG expansions

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  • Опубліковано 13 чер 2024
  • This edition of the Research Video Series features the HD Buzz article, Two Birds One Stone. In a surprising twist, oral HTT-lowering drugs also slow somatic expansion in the HTT gene. A new study that used cells in a dish for this fortuitous discovery identified the gene PMS1 as a key player in the slowing of CAG expansions.
    Read the entire HD Buzz article, en.hdbuzz.net/367
    The Research Video Series is sponsored by Roche, Sage Therapeutics, Teva Pharmaceutical, uniQure, Wave Life Sciences, Griffin Foundation and Neurocrine Biosciences.

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