Safety and efficacy of RNAi therapy for transthyretin amyloidosis.

@article{Coelho2013SafetyAE,
  title={Safety and efficacy of RNAi therapy for transthyretin amyloidosis.},
  author={Teresa Coelho and David H Adams and Ana Gabriela Aguilera Silva and Pierre Lozeron and Philip N. Hawkins and T. S. K. Mant and Javier Serrano P{\'e}rez and Joseph A Chiesa and Steve Warrington and Elizabeth Tranter and Malathy Munisamy and Rick Falzone and James S. Harrop and Jeffrey E. Cehelsky and Brian R. Bettencourt and Mary Geissler and James Scott Butler and Alfica Sehgal and Rachel Ellen Meyers and Qingmin Chen and Todd Douglass Borland and Renta M. Hutabarat and Valerie A. Clausen and Ren{\'e} Cece{\~n}a {\'A}lvarez and Kevin C Fitzgerald and Christina Gamba-Vitalo and Saraswathy V. Nochur and Akshay K. Vaishnaw and Dinah W. Y. Sah and Jared A. Gollob and Ole Bernt Suhr},
  journal={The New England journal of medicine},
  year={2013},
  volume={369 9},
  pages={
          819-29
        }
}
BACKGROUND Transthyretin amyloidosis is caused by the deposition of hepatocyte-derived transthyretin amyloid in peripheral nerves and the heart. A therapeutic approach mediated by RNA interference (RNAi) could reduce the production of transthyretin. METHODS We identified a potent antitransthyretin small interfering RNA, which was encapsulated in two distinct first- and second-generation formulations of lipid nanoparticles, generating ALN-TTR01 and ALN-TTR02, respectively. Each formulation was… CONTINUE READING
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