CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis.

  title={CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis.},
  author={Julian D. Gillmore and E. H. Gane and Jorg Taubel and Justin C. Kao and Marianna Fontana and Michael L. Maitland and Jessica Seitzer and Daniel O’Connell and Kathryn R Walsh and Kristy Wood and Jonathan Phillips and Yuanxin Xu and Adam Amaral and Adam P. Boyd and Jeffrey E Cehelsky and Mark McKee and Andrew Schiermeier and Olivier A. Harari and Andrew Murphy and Christos A. Kyratsous and Brian Zambrowicz and Randy Soltys and David E. Gutstein and John Leonard and Laura Sepp-Lorenzino and David Lebwohl},
  journal={The New England journal of medicine},
BACKGROUND Transthyretin amyloidosis, also called ATTR amyloidosis, is a life-threatening disease characterized by progressive accumulation of misfolded transthyretin (TTR) protein in tissues, predominantly the nerves and heart. NTLA-2001 is an in vivo gene-editing therapeutic agent that is designed to treat ATTR amyloidosis by reducing the concentration of TTR in serum. It is based on the clustered regularly interspaced short palindromic repeats and associated Cas9 endonuclease (CRISPR-Cas9… 

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