CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

Julian D. Gillmore,Ed Gane,Jorg Taubel,Justin Kao,Marianna Fontana,Michael L. Maitland,Jessica Seitzer,Daniel J. Corporal Oconnell,Kathryn R. Walsh,Wood Kristy M,Jonathan Phillips,Yuanxin Xu,Adam Amaral,Adam P. Boyd,Jeffrey Cehelsky,Mark D. McKee,Andrew Schiermeier,Olivier Harari,Andrew J. Murphy,Christos Kyratsous,Brian Zambrowicz,Randy Soltys,David E. Gutstein,John P. Leonard,Laura Sepp-Lorenzino,David Lebwohl

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

2021

Abstract Background Transthyretin amyloidosis, also called ATTR amyloidosis, is a life-threatening disease characterized by progressive accumulation of misfolded transthyretin (TTR) protein in tiss...

Keywords:

In vivo
Transthyretin
Medicine
multicenter study
attr amyloidosis
CRISPR
Amyloidosis
Genome editing
Cancer research

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