Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations
John K. Yoon(ProterixBio (United States)), Niek P. van Til(Amsterdam Neuroscience), Richard W. Pfeifer(ProterixBio (United States)), Robert N. Plasschaert(ProterixBio (United States)), Yildirim Dogan(Memorial Sloan Kettering Cancer Center), Claudia Fiorini(Albert Einstein College of Medicine), Zeenath Unnisa(ProterixBio (United States)), Fritz Hull(ProterixBio (United States)), Jeffrey W. Schindler(Forma Therapeutics (United States)), Luigi Barbarossa(ProterixBio (United States)), Luca Biasco(Institute of Infection and Immunity), Daniel Ivanov(ProterixBio (United States)), Axel Schambach(Boston Children's Hospital), Mariana Loperfido(ProterixBio (United States)), Cristina Baricordi(Broad Institute), Robert Kutner(ProterixBio (United States)), Swaroopa Guda(ProterixBio (United States)), Tim Maiwald(ProterixBio (United States)), Mark P. DeAndrade(ProterixBio (United States)), Christine Oborski(ProterixBio (United States)), Mary E. Jacobs(ProterixBio (United States)), Véronique Michaud(Research & Development Institute), Michael Rothe(American Society of Clinical Oncology), Aimin Yan(ProterixBio (United States)), Christopher M. Treleaven(ProterixBio (United States)), Cecilia Barese(ProterixBio (United States)), Chris Mason(Oak Ridge National Laboratory), Vicky Ping Chen(ProterixBio (United States))
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