Article
Gene therapy for hemophilia: Current status and laboratory consequences.
International journal of laboratory hematology - 1 Jul 2021
Batty Paul, Lillicrap David
Abstract excerpt
Since the cloning and characterization of the factor VIII (FVIII) and factor IX genes in the mid-1980s, gene therapy has been perceived as having significant potential for the treatment of severe hemophilia. Now, some 35 years later, these proposals are close to being realized through the licensing of the first clinical gene therapy product. Adeno-associated viral vector-mediated gene therapy for hemophilia A and...
Topics
- Animals
- Blood Coagulation
- Blood Coagulation Tests
- Clinical Trials as Topic
- Dependovirus
- Factor IX
- Factor VIII
- Genetic Therapy
- Genetic Vectors
- Hemophilia A
- Hemophilia B
- Humans