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Molecular evaluation and vector integration analysis of HCC complicating AAV gene therapy for hemophilia B

Clinical Trials & Observations
Manfred Schmidt, Graham R. Foster, Michiel Coppens, Hauke Thomsen, Ricardo Dolmetsch, Liesbeth Heijink, Paul E. Monahan, Steven W. Pipe
Journal: Blood Advances
Blood Adv (2023) 7 (17): 4966–4969.
https://doi.org/10.1182/bloodadvances.2023009876
Published: 2023
Includes: Supplemental data