Gene Therapy for Hemophilias: Opportunities and Challenges for the Clinical Laboratory

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Gene therapy effectively sustains plasma levels of Factor VIII and IX, reducing bleeding incidents in patients.
Transgene factors are measurable by one-stage clotting and chromogenic assays, with OSA showing about double levels of CA.
Adeno-associated viral vectors are utilized to transfer relevant gene material into liver cells for producing Factor VIII or IX.
Selecting patients without liver disease is critical for ensuring successful gene therapy outcomes.

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Clinical trials show gene therapy improves Factor VIII and IX levels in hemophilia, suggesting a shift in treatment strategy.

Tripodi et al. (2025) studied this question.

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