Adeno-associated viral vector integration: implications for long-term efficacy and safety.

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第一作者： Paul,Batty

第一单位： Department of Haematology, Cancer Institute, University College London, London, United Kingdom; Department of Pathology and Molecular Medicine, Queen's University, Kingston, Ontario, Canada. Electronic address: paul.batty@ucl.ac.uk.

作者： Paul,Batty ^[1] ; David,Lillicrap ^[2]

作者单位： Department of Haematology, Cancer Institute, University College London, London, United Kingdom; Department of Pathology and Molecular Medicine, Queen's University, Kingston, Ontario, Canada. Electronic address: paul.batty@ucl.ac.uk. ^[1] Department of Pathology and Molecular Medicine, Queen's University, Kingston, Ontario, Canada. ^[2]

关键词 adeno-associated virus episome gene therapy hemophilia A virus integration

主题词人类(Humans);依赖病毒(Dependovirus);遗传载体(Genetic Vectors);动物(Animals);病毒整合(Virus Integration);血友病A(Hemophilia A);治疗结果(Treatment Outcome)

DOI 10.1016/j.jtha.2024.07.012

PMID 39097231

发布时间 2024-10-27

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