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

作者：Manfred Schmidt, Graham R. Foster, Michiel Coppens, Hauke Thomsen, Ricardo Dolmetsch, Liesbeth Heijink, Paul E. Monahan, Steven W. Pipe · 发表于：Blood Advances · 年份：2023 · DOI：10.1182/bloodadvances.2023009876 · 被引用次数：57 · 研究领域：Virus-based gene therapy research、Viral Infectious Diseases and Gene Expression in Insects、RNA Interference and Gene Delivery