CRISPR-Cas9 in vivo gene editing for transthyretin amyloidosis
Julian D. Gillmore, Ed Gane, Jörg Täubel, Justin Kao, Marianna Fontana, Michael L. Maitland, Jessica Seitzer, Daniel O’Connell, Kathryn Walsh, Kristy Wood, Jonathan D. Phillips, Yuanxin Xu, Adam Amaral, Adam P. Boyd, Jeffrey Cehelsky, Mark D. McKee, Andrew Schiermeier, Olivier Harari, Andrew Murphy, Christos A. Kyratsous, Brian Zambrowicz, Randy Soltys, David E. Gutstein, John P. Leonard, Laura Sepp‐Lorenzino, David Lebwohl
Abstract
N Engl J Med. 2021 Aug 5;385(6):493–502. PMID: 34215024 doi: 10.1056/NEJMoa2107454. Epub 2021 Jun 26.
Topics & Concepts
CRISPRGenome editingTransthyretinComputational biologyIn vivoGeneBiologyGeneticsEndocrinologyCRISPR and Genetic Engineering