The New England journal of medicine, 2017-12, Vol.377 (23), p.2215-2227
- 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
2017
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Details
- Autor(en) / Beteiligte
- 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
- Titel
- Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant
- Ist Teil von
- The New England journal of medicine, 2017-12, Vol.377 (23), p.2215-2227
- Ort / Verlag
- United States: Massachusetts Medical Society
- Erscheinungsjahr
- 2017
- Quelle
- MEDLINE
- Beschreibungen/Notizen
- 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.
- Sprache
- Englisch
- Identifikatoren
- ISSN: 0028-4793eISSN: 1533-4406DOI: 10.1056/NEJMoa1708538Titel-ID: cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_6029626
- Format
- –
- 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