Recent Advances in Gene Therapy for Hemophilia.

ABSTRACT: Hemophilia, an X-linked monogenic disorder, arises from mutations in the F8 or F9 genes, which encode clotting factor VIII (FVIII) or clotting factor IX (FIX), respectively. As a prominent hereditary coagulation disorder, hemophilia is clinically manifested by spontaneous hemorrhagic episodes. Severe cases may progress to complications such as stroke and arthropathy, significantly compromising patients' quality of life. Hemophilia has a monogenic nature, coupled with quantifiable therapeutic endpoints and predictable treatment outcomes. These characteristics render it an ideal candidate for gene therapy studies. Currently, Food and Drug Administration (FDA)-approved gene therapies utilize recombinant adeno-associated virus (AAV) vectors to deliver functional transgene cassett

SUBMITTER: Pang X 

PROVIDER: S-EPMC12423543 | biostudies-literature | 2025 Jan-Dec

REPOSITORIES: biostudies-literature