High-resolution cellular and molecular follow up of lysosomal disease patients treated with hematopoietic stem cell lentiviral gene therapy
Mariana Loperfido(ProterixBio (United States)), Luca Biasco(Institute of Infection and Immunity), Quoc Nguyen(ProterixBio (United States)), Luigi Barbarossa(ProterixBio (United States)), Stéphanie Cherqui(University of California San Diego), Cristina Baricordi(Broad Institute), Aimin Yan(ProterixBio (United States)), Ingrid Abrahamsen(ProterixBio (United States)), Chris Mason(Oak Ridge National Laboratory), Diana M. Escolar(Children's National)
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