Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vectorMaria Dahl, Stefan Karlsson, Axel Schambach et al.|Molecular Therapy — Methods & Clinical Development|2020Cited by 23
AVR-RD-01 lentiviral gene therapy reduces Gb3 substrate in endothelial cells of renal peritubular capillaries in a previously untreated classic Fabry disease male patientBirgitte Volck, Chris Mason, Shari Fallet et al.|Molecular Genetics and Metabolism|2020Cited by 2
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 diseaseLeslie K. Jacobsen, Chris Mason, Julie A. Kerner et al.|Molecular Genetics and Metabolism|2021Cited by 1
A Comprehensive High Resolution Scrna-Seq Analytical Platform for Characterizing Human Hematopoietic Stem/Progenitor Cells States in Ex Vivo Drug ProductsCristina Baricordi, Luca Biasco, Mariana Loperfido et al.|Blood|2020Cited by 0
High throughput monitoring of safety, potency and stability of gene therapy cell products in lysosomal disease patientsLuca Biasco, Chris Mason, Cristina Baricordi et al.|Molecular Genetics and Metabolism|2022Cited by 0