Open AccessMultiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A
Author(s) -
John Pasi,
Savita Rangarajan,
Nina Mitchell,
Will Lester,
Emily Symington,
Bella Madan,
Michael Laffan,
Chris B. Russell,
Mingjin Li,
Glenn F. Pierce,
Wing Yen Wong
Publication year - 2020
Publication title -
new england journal of medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 19.889
H-Index - 1030
eISSN - 1533-4406
pISSN - 0028-4793
DOI - 10.1056/nejmoa1908490
Subject(s) - medicine , factor ix , body weight , genetic enhancement , gastroenterology , surgery , gene , biochemistry , chemistry
Adeno-associated virus (AAV)-mediated gene therapy is under investigation as a therapeutic option for persons with hemophilia A. Efficacy and safety data include 3 years of follow-up after a single administration of AAV5-hFVIII-SQ.
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