Article
New approaches to gene and cell therapy for hemophilia.
Journal of thrombosis and haemostasis : JTH - 1 Jun 2015
Ohmori T, Mizukami H, Ozawa K, Sakata Y, Nishimura S
Abstract excerpt
Hemophilia is considered suitable for gene therapy because it is caused by a single gene abnormality, and therapeutic coagulation factor levels may vary across a broad range. Recent success of hemophilia B gene therapy with an adeno-associated virus (AAV) vector in a clinical trial showed the real prospect that, through gene therapy, a cure for hemophilia may become a reality. However, AAV-mediated gene therapy...
Topics
- Animals
- Blood Coagulation
- Dependovirus
- Gene Transfer Techniques
- Genetic Predisposition to Disease
- Genetic Therapy
- Genetic Vectors
- Hemophilia A
- Hemorrhage
- Humans
- Phenotype