Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

Nathwani, Amit; M. Reiss, Ulreke; G. D. Tuddenham, Edward; Rosales, Cecilia; Chowdary, Pratima; McIntosh, Jenny; Della Peruta, Marco; Lhériteau, Elsa; Patel, Nishal; B. Thimiri Govinda Raj, Deepak; Riddell, Anne; Pie, Jun; Rangarajan, Savita; Bevan, David; Recht, Michael; Shen, Yu‐Min; G. Halka, Kathleen; Basner‐Tschakarjan, Etiena; Mingozzi, Federico; A. High, Katherine; A. Allay, James; A. Kay, Mark; Y. Ng, Catherine; Zhou, Junfang; Cancio, Maria; L. Morton, Christopher; T. Gray, John; Kumar Srivastava, Deo; W. Nienhuis, Arthur; M. Davidoff, Andrew

doi:10.1056/nejmoa1407309

Public

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

Published in New England Journal of Medicine • Nov 20, 2014

Authors:

Amit Nathwani

Ulreke M. Reiss

Edward G. D. Tuddenham

Abstract

BACKGROUND: In patients with severe hemophilia B, gene therapy that is mediated by a novel self-complementary adeno-associated virus serotype 8 (AAV8) vector has been shown to raise factor IX levels for periods of up to 16 months. We wanted to determine the durability of transgene expression, the ve...

Subject

Factor IX

Medicine

Genetic enhancement

Research Assistant

AI chat, annotations, notes & similar papers

Finding related papers...

Discussions

(0)

No comments yet

Be the first to share your thoughts!