Screening of Chimeric GAA Variants in a Preclinical Study of Pompe Disease Results in Candidate Vector for Hematopoietic Stem Cell Gene Therapy
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)), 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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