Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation.

@article{Herzog2001MuscledirectedGT,
  title={Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation.},
  author={Roland W Herzog and Jane D. Mount and Valder R Arruda and Katherine A High and Clinton D. Lothrop},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2001},
  volume={4 3},
  pages={
          192-200
        }
}
The X-linked bleeding disorder hemophilia B is caused by absence of functional blood coagulation factor IX (F9) and can be treated by adeno-associated viral (AAV) mediated gene transfer to skeletal muscle. The safety of this approach is currently being evaluated in a phase I clinical trial. Efficacy of this and several other gene therapy strategies has been addressed in hemophilia B dogs, an important preclinical model of the disease. While previously published data demonstrated sustained… CONTINUE READING
BETA

Similar Papers

Citations

Publications citing this paper.
SHOWING 1-10 OF 85 CITATIONS, ESTIMATED 52% COVERAGE

Translational data from adeno-associated virus-mediated gene therapy of hemophilia B in dogs.

  • Human gene therapy. Clinical development
  • 2015
VIEW 4 EXCERPTS
CITES BACKGROUND
HIGHLY INFLUENCED

FILTER CITATIONS BY YEAR

2002
2019

CITATION STATISTICS

  • 4 Highly Influenced Citations