Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant

An adeno-associated viral vector was used to introduce a FIX gene with enhanced biologic activity in 10 participants with hemophilia B. The annualized bleeding rate was 11.1 events per year before therapy versus 0.4 afterward. Steady-state factor IX levels were 33.7% of normal.

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Veröffentlicht in:	The New England journal of medicine 2017-12, Vol.377 (23), p.2215-2227
Hauptverfasser:	George, Lindsey A, Sullivan, Spencer K, Giermasz, Adam, Rasko, John E.J, Samelson-Jones, Benjamin J, Ducore, Jonathan, Cuker, Adam, Sullivan, Lisa M, Majumdar, Suvankar, Teitel, Jerome, McGuinn, Catherine E, Ragni, Margaret V, Luk, Alvin Y, Hui, Daniel, Wright, J. Fraser, Chen, Yifeng, Liu, Yun, Wachtel, Katie, Winters, Angela, Tiefenbacher, Stefan, Arruda, Valder R, van der Loo, Johannes C.M, Zelenaia, Olga, Takefman, Daniel, Carr, Marcus E, Couto, Linda B, Anguela, Xavier M, High, Katherine A
Format:	Artikel
Sprache:	eng
Schlagworte:	Adolescent Adult Bleeding Body weight Clotting Coagulation factors Dependovirus - immunology Drug dosages Factor IX - genetics Factor IX - metabolism Factor IX - therapeutic use Factor IX deficiency Gene therapy Genetic Therapy - methods Genetic Vectors - administration & dosage Genomes Hemophilia Hemophilia B - genetics Hemophilia B - metabolism Hemophilia B - therapy Hemorrhage - prevention & control Humans Liver Male Middle Aged Prednisone Prophylaxis Transgenes Young Adult
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Beschreibung
Zusammenfassung:	An adeno-associated viral vector was used to introduce a FIX gene with enhanced biologic activity in 10 participants with hemophilia B. The annualized bleeding rate was 11.1 events per year before therapy versus 0.4 afterward. Steady-state factor IX levels were 33.7% of normal.
ISSN:	0028-4793 1533-4406
DOI:	10.1056/NEJMoa1708538