Evaluating Gene Therapy as a Potential Paradigm Shift in Treating Severe Hemophilia.

ABSTRACT: Hemophilia is characterized by a deficiency in coagulation factors VIII or IX. The general standard of care for severe hemophilia is frequent intravenous recombinant or plasma-derived factor replacement to prevent bleeding. While this treatment is effective in preventing bleeding, frequent infusions are burdensome for patients. Nonadherence to the therapeutic regimen leaves people with hemophilia at risk for spontaneous and traumatic bleeds into joints as well as life-threatening bleeds such as intracranial hemorrhage. The chronicity of the disorder often leads to the formation of target joints, causing long-term pain and impairing mobility. As a monogenic disorder with well-understood genetics, hemophilia is an ideal disorder for implementing innovations in gene therapies. Indeed, recent

SUBMITTER: Thornburg CD 

PROVIDER: S-EPMC10432364 | biostudies-literature | 2023 Sep

REPOSITORIES: biostudies-literature