Article
Bioengineering hemophilia A-specific microvascular grafts for delivery of full-length factor VIII into the bloodstream.
Blood advances - 23 Dec 2019
Neumeyer Joseph, Lin Ruei-Zeng, Wang Kai, Hong Xuechong, Hua Tien, Croteau Stacy E, Neufeld Ellis J, Melero-Martin Juan M
Abstract excerpt
Hemophilia A (HA) is a bleeding disorder caused by mutations in the F8 gene encoding coagulation factor VIII (FVIII). Current treatments are based on regular infusions of FVIII concentrates throughout a patient's life. Alternatively, viral gene therapies that directly deliver F8 in vivo have shown preliminary successes. However, hurdles remain, including lack of infection specificity and the inability to deliver...
Topics
- Animals
- Bioengineering
- Blood Coagulation
- Blood Coagulation Tests
- Disease Models, Animal
- Embryonic Stem Cells
- Factor VIII
- Gene Expression
- Gene Transfer Techniques
- Genetic Therapy
- Hemophilia A
- Humans
- Induced Pluripotent Stem Cells