Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector

Maria Dahl(Lund University), Stefan Karlsson(Kristianstad University), Johannes M. F. G. Aerts(Leiden University Medical Center), Richard W. Pfeifer(ProterixBio (United States)), Maria J. Ferraz(Amsterdam UMC Location University of Amsterdam), Axel Schambach(Boston Children's Hospital), Emma Smith(Lund University), Sarah Warsi(Lund University), Azadeh Golipour(ProterixBio (United States)), Michael Rothe(American Society of Clinical Oncology), Claudia Harper(ProterixBio (United States)), Chris Mason(Oak Ridge National Laboratory), Daniella Pizzurro(ProterixBio (United States))
Molecular Therapy — Methods & Clinical Development
December 2, 2020
Cited by 23


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