Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies

Laura Breda(Children's Hospital of Philadelphia), Stefano Rivella(Children's Hospital of Philadelphia), J.K. Everett(University of Pennsylvania), Enrico Radaelli(University of Pennsylvania), Frederic D. Bushman(University of Pennsylvania), Carla Casu(Children's Hospital of Philadelphia), Xiaochuan Shan(University of Pennsylvania), Danuta Jarocha(Jagiellonian University), Virginia Guzikowski(Children's Hospital of Philadelphia), Ryo Kurita(Japanese Red Cross Society, Japan), Natalie Hoepp(University of Pennsylvania), Valentina Ghiaccio(Children's Hospital of Philadelphia), Naoto Tanaka(Temple College), Yukio Nakamura(Kyorin University), Osheiza Abdulmalik(Children's Hospital of Philadelphia), Tobias Raabe(University of Pennsylvania), Alisa Dong(Children's Hospital of Philadelphia), Gwenn Danet-Desnoyers(University of Pennsylvania), Janet L. Kwiatkowski(Children's Hospital of Philadelphia), Kim Smith‐Whitley(Children's Hospital of Philadelphia), James C. Tarrant(University of Pennsylvania), Yasuhiro Ikawa(Children's Hospital of Philadelphia), Charles A. Assenmacher(University of Pennsylvania), Aoife Doto(University of Pennsylvania)
Molecular Therapy
January 29, 2021
Cited by 23


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