Nature Communications (Feb 2021)

Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models

  • Yuanjing Liu,
  • Jean-Cosme Dodart,
  • Helene Tran,
  • Shaunna Berkovitch,
  • Maurine Braun,
  • Michael Byrne,
  • Ann F. Durbin,
  • Xiao Shelley Hu,
  • Naoki Iwamoto,
  • Hyun Gyung Jang,
  • Pachamuthu Kandasamy,
  • Fangjun Liu,
  • Kenneth Longo,
  • Jörg Ruschel,
  • Juili Shelke,
  • Hailin Yang,
  • Yuan Yin,
  • Amy Donner,
  • Zhong Zhong,
  • Chandra Vargeese,
  • Robert H. Brown

DOI
https://doi.org/10.1038/s41467-021-21112-8
Journal volume & issue
Vol. 12, no. 1
pp. 1 – 15

Abstract

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C9orf72 expansion mutations are the most common genetic cause of ALS and FTD, which have limited therapies. The authors generate stereopure oligonucleotides that selectively deplete expansion-containing transcripts and protect against expansion-associated pathologies in preclinical models.