AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B
Children's Hospital of Philadelphia · Bayer (United States) · +6 more institutions
Abstract
Hemophilia B is an X-linked coagulopathy caused by absence of functional coagulation factor IX (F.IX). Previously, we established an experimental basis for gene transfer as a method of treating the disease in mice and hemophilic dogs through intramuscular injection of a recombinant adeno-associated viral (rAAV) vector expressing F.IX. In this study we investigated the safety of this approach in patients with hemophilia B. In an open-label dose-escalation study, adult men with severe hemophilia B (F.IX
Citation impact
- FWCI
- 32.72
- Percentile
- 100%
- References
- 78
Authors
20- CSCatherine S. MannoCorresponding
Children's Hospital of Philadelphia, Bayer (United States), University of Pittsburgh, Universidade Estadual de Campinas (UNICAMP), Bloodworks Northwest, Hemophilia Center of Western Pennsylvania, University of Pennsylvania, Stanford University
- AKAh Keng Chew
Children's Hospital of Philadelphia, Bayer (United States), University of Pittsburgh, Universidade Estadual de Campinas (UNICAMP), Bloodworks Northwest, Hemophilia Center of Western Pennsylvania, University of Pennsylvania, Stanford University
- SHS Hutchison
Children's Hospital of Philadelphia, Bayer (United States), University of Pittsburgh, Universidade Estadual de Campinas (UNICAMP), Bloodworks Northwest, Hemophilia Center of Western Pennsylvania, University of Pennsylvania, Stanford University
- PJPeter J. Larson
Children's Hospital of Philadelphia, Bayer (United States), University of Pittsburgh, Universidade Estadual de Campinas (UNICAMP), Bloodworks Northwest, Hemophilia Center of Western Pennsylvania, University of Pennsylvania, Stanford University
- RWRoland W. Herzog
Children's Hospital of Philadelphia, Bayer (United States), University of Pittsburgh, Universidade Estadual de Campinas (UNICAMP), Bloodworks Northwest, Hemophilia Center of Western Pennsylvania, University of Pennsylvania, Stanford University
Topics & keywords
- Factor IX
- Genetic enhancement
- Adeno-associated virus
- Medicine
- Intramuscular injection
- Coagulopathy
- Recombinant DNA
- Virology
- Good health and well-being