AVR-RD-01 lentiviral gene therapy reduces Gb3 substrate in endothelial cells of renal peritubular capillaries in a previously untreated classic Fabry disease male patient

Birgitte Volck(ProterixBio (United States)), Chris Mason(Oak Ridge National Laboratory), Dawn Phillips(ProterixBio (United States)), B. Carnley(Royal Perth Hospital), Mark Thomas(Royal Perth Hospital), Azadeh Golipour(ProterixBio (United States)), Shari Fallet(ProterixBio (United States))
Molecular Genetics and Metabolism
January 30, 2020
Cited by 2


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