CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis.
N Engl J Med. 2021 Aug 5;385(6):493-502. doi: 10.1056/NEJMoa2107454. Epub 2021 Jun 26.
N Engl J Med. 2021.
PMID: 34215024
Clinical Trial.