Screening chimeric GAA variants in preclinical study results in hematopoietic stem cell gene therapy candidate vectors for Pompe disease
Yildirim Dogan(Memorial Sloan Kettering Cancer Center), Niek P. van Til(Amsterdam Neuroscience), Richard W. Pfeifer(ProterixBio (United States)), Zeenath Unnisa(ProterixBio (United States)), Jeffrey W. Schindler(Forma Therapeutics (United States)), Axel Schambach(Boston Children's Hospital), John K. Yoon(ProterixBio (United States)), Swaroopa Guda(ProterixBio (United States)), Diana L. Clarke(ProterixBio (United States)), Tim Maiwald(ProterixBio (United States)), Christine Oborski(ProterixBio (United States)), Mary E. Jacobs(ProterixBio (United States)), Claudia Harper(ProterixBio (United States)), Chris Mason(Oak Ridge National Laboratory), Cecilia Barese(ProterixBio (United States))
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