CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

N Engl J Med. 2021 Oct 28;385(18):1722. doi: 10.1056/NEJMc2114592.
No abstract available

Publication types

  • Letter
  • Comment

MeSH terms

  • Amyloid Neuropathies, Familial* / genetics
  • Amyloid Neuropathies, Familial* / therapy
  • CRISPR-Cas Systems
  • Gene Editing*
  • Humans
  • RNA, Guide, CRISPR-Cas Systems

Substances

  • RNA, Guide, CRISPR-Cas Systems

Supplementary concepts

  • Amyloidosis, Hereditary, Transthyretin-Related