The GuardOne clinical trial: A first-in-human, open-label, multinational phase 1/2 study of AVR-RD-02 ex vivo lentiviral vector, autologous gene therapy for Gaucher disease

Leslie K. Jacobsen(Johns Hopkins University), Chris Mason(Oak Ridge National Laboratory), Julie A. Kerner, Josie Yang, Azadeh Golipour(ProterixBio (United States)), Rachel Ciotti, Richard W. Pfeifer(ProterixBio (United States))
Molecular Genetics and Metabolism
February 1, 2021
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