Disease Score gda Association Type Type Original DB Sentence supporting the association PMID PMID Year
CUI: C0019069
Disease: Hemophilia A
Hemophilia A
0.060 GeneticVariation disease BEFREE Thus, AAV gene therapies are likely to alter the treatment paradigm for hemophilia A and B. 31808868 2019
CUI: C0019069
Disease: Hemophilia A
Hemophilia A
0.060 GeneticVariation disease BEFREE Despite the initial success of liver-directed adeno-associated virus (AAV) gene therapy for hemophilia in clinical trials, long-term sustained therapeutic effects have yet to be seen. 30975639 2019
CUI: C0019069
Disease: Hemophilia A
Hemophilia A
0.060 Biomarker disease BEFREE Here, we provide an overview of the clinical development of AAV gene transfer for hemophilia, as well as an outlook on future directions. 28411016 2017
CUI: C0019069
Disease: Hemophilia A
Hemophilia A
0.060 Biomarker disease BEFREE Adeno-associated virus (AAV) gene therapy vectors have shown the best outcomes in human clinical studies for the treatment of genetic diseases such as hemophilia. 28460646 2017
CUI: C0019069
Disease: Hemophilia A
Hemophilia A
0.060 Biomarker disease BEFREE Adeno-associated viral (AAV) gene transfer of coagulation factor IX to skeletal muscle and liver of murine and canine models of hemophilia has resulted in sustained systemic expression and, in several studies, in complete cure of the bleeding disorder. 17266422 2007
CUI: C0019069
Disease: Hemophilia A
Hemophilia A
0.060 Biomarker disease BEFREE Adeno-associated viral (AAV) gene transfer of coagulation factor VIII and IX to skeletal muscle and liver of murine and canine models of hemophilia A and B have resulted in sustained systemic expression and, in several studies, in complete cure of the bleeding disorder. 15975012 2005