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Gene Therapy for Hemophilia-Opportunities and Risks

Wolfgang Miesbach1, Robert Klamroth, Johannes Oldenburg

  • 1Department of Haemostaseology and Hemophilia Center, Medical Clinic 2, Institute of Transfusion Medicine, University Hospital Frankfurt, Frankfurt am Main; Department for Internal Medicine, Vascular Medicine and Haemostaseology, Vivantes Klinikum im Friedrichshain, Berlin; Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn; Department of Hematology, Hemostaseology, Oncology and Stem Cell Transplantation, Hannover Medical School.

Deutsches Arzteblatt International
|December 5, 2022
PubMed
Summary

Adeno-associated virus (AAV)-based gene therapy offers a promising, one-time treatment for hemophilia, significantly reducing bleeding episodes. While effective, potential antibody formation and inflammatory responses require careful management.

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