@article{discovery10197724, journal = {Molecular Therapy}, year = {2024}, title = {Preclinical lentiviral vector-mediated hematopoietic stem and progenitor cell gene therapy corrects Pompe disease-related muscle and neurological manifestations}, month = {September}, publisher = {Nature Publishing Group}, note = {This version is the author accepted manuscript. For information on re-use, please refer to the publisher's terms and conditions.}, author = {Yoon, John K and Schindler, Jeffrey W and Loperfido, Mariana and Baricordi, Cristina and DeAndrade, Mark P and Jacobs, Mary E and Treleaven, Christopher and Plasschaert, Robert N and Yan, Aimin and Barese, Cecilia N and Dogan, Yildirim and Chen, Vicky Ping and Fiorini, Claudia and Hull, Fritz and Barbarossa, Luigi and Unnisa, Zeenath and Ivanov, Daniel and Kutner, Robert H and Guda, Swaroopa and Oborski, Christine and Maiwald, Tim and Michaud, V{\'e}ronique and Rothe, Michael and Schambach, Axel and Pfeifer, Richard and Mason, Chris and Biasco, Luca and van Til, Niek P}, url = {http://dx.doi.org/10.1016/j.ymthe.2024.09.024} }