ESPE Yearbook of Paediatric Endocrinology

ESPE Yearbook of Paediatric Endocrinology

ISSN 1662-4009 (online)

Published by BioScientifica

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ey0019.14-13 | New treatments | ESPEYB19

14.13. CRISPR-Cas9 in vivo gene editing for transthyretin amyloidosis

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

N Engl J Med. 2021 Aug 5;385(6):493–502. PMID: 34215024 doi: 10.1056/NEJMoa2107454. Epub 2021 Jun 26.Brief Summary: 6 patients with hereditary, life-threatening transthyretin amyloidosis were treated with a novel in vivo gene-editing therapeutic agent (NTLA-2001) based on CRISPR-Cas9 technology. The new agent was given intravenously in a single dose and was able to durably knockou...
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Published by BioScientifica

ESPE Yearbook of Paediatric Endocrinology
ISSN 1662-4009 (Online)
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