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Comment
. 2017 Dec 14;171(7):1478-1480.
doi: 10.1016/j.cell.2017.11.049.

FIX It in One Go: Enhanced Factor IX Gene Therapy for Hemophilia B

David Lillicrap  1
Affiliations
Free article
Comment

FIX It in One Go: Enhanced Factor IX Gene Therapy for Hemophilia B

David Lillicrap. Cell. .
Free article

Abstract

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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  • Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.
    George LA, Sullivan SK, Giermasz A, Rasko JEJ, Samelson-Jones BJ, Ducore J, Cuker A, Sullivan LM, Majumdar S, Teitel J, McGuinn CE, Ragni MV, Luk AY, Hui D, Wright JF, Chen Y, Liu Y, Wachtel K, Winters A, Tiefenbacher S, Arruda VR, van der Loo JCM, Zelenaia O, Takefman D, Carr ME, Couto LB, Anguela XM, High KA. George LA, et al. N Engl J Med. 2017 Dec 7;377(23):2215-2227. doi: 10.1056/NEJMoa1708538. N Engl J Med. 2017. PMID: 29211678 Free PMC article. Clinical Trial.

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