Long-term hematopoietic stem cell gene therapy corrects neuromuscular manifestations in preclinical study of Pompe mice

Publication date: February 2021Source: Molecular Genetics and Metabolism, Volume 132, Issue 2Author(s): Niek P. van Til, Yildirim Dogan, Cecilia Barese, Zeenath Unnisa, Swaroopa Guda, Rena Schindler, John Yoon, Mary Jacobs, Abhishek Chiyyeadu, Daniella Pizzurro, Bianling Liu, Maurine Braun, Christine Oborski, Axel Schambach, Claudia Harper, Richard Pfeifer, Chris Mason
Source: Molecular Genetics and Metabolism - Category: Genetics & Stem Cells Source Type: research