Open AccessFIX It in One Go: Enhanced Factor IX Gene Therapy for Hemophilia B
Author(s) -
David Lillicrap
Publication year - 2017
Publication title -
cell
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 26.304
H-Index - 776
eISSN - 1097-4172
pISSN - 0092-8674
DOI - 10.1016/j.cell.2017.11.049
Subject(s) - biology , factor ix , genetic enhancement , gene , genetics , virology , computational biology
A phase 1/2 clinical trial of AAV-mediated gene therapy in patients with hemophilia B using an enhanced specific activity factor IX (FIX) transgene reports sustained levels of FIX levels, leading to the near elimination of bleeding for more than a year and without serious adverse side effects. These results are the best outcome to date for hemophilia gene therapy.
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