In vivo CRISPR/Cas9 editing of the TTR gene with NTLA-2001 in patients with transthyretin amyloidosis- dose selection considerations
Edward Gane(University of Auckland), Gillmore Julian(University College London), Jonathan D. Phillips(University of Kentucky), Kristy Wood(University of Auckland), Michael L. Maitland(Virginia Cancer Institute), Laura Sepp‐Lorenzino(Intellia Therapeutics (United States)), Adam Amaral(University of Auckland), David Lebwohl(Intellia Therapeutics (United States)), Mark Stroh(CytomX Therapeutics (United States)), Odelya E. Pagovich(Regeneron (United States)), Carri Boiselle(Amsterdam University Medical Centers), Jörg Täubel(British Heart Foundation), David Gustein(Regeneron (United States)), Yuanxin Xu(Intellia Therapeutics (United States)), Marianna Fontana(The Royal Free Hospital), Adam P. Boyd(University of Auckland), Justin Kao(University of Auckland), Liron Walsh(Pediatric Nephrology of Alabama), Jessica Seitzer(Intellia Therapeutics (United States)), Jeffrey Cehelsky(Alnylam Pharmaceuticals (United States)), Björn Pilebro(Umeå University)
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