Yoon, John K;    Schindler, Jeffrey W;    Loperfido, Mariana;    Baricordi, Cristina;    DeAndrade, Mark P;    Jacobs, Mary E;    Treleaven, Christopher;                                                                                     ... van Til, Niek P; + view all <#>        Yoon, John K;  Schindler, Jeffrey W;  Loperfido, Mariana;  Baricordi, Cristina;  DeAndrade, Mark P;  Jacobs, Mary E;  Treleaven, Christopher;  Plasschaert, Robert N;  Yan, Aimin;  Barese, Cecilia N;  Dogan, Yildirim;  Chen, Vicky Ping;  Fiorini, Claudia;  Hull, Fritz;  Barbarossa, Luigi;  Unnisa, Zeenath;  Ivanov, Daniel;  Kutner, Robert H;  Guda, Swaroopa;  Oborski, Christine;  Maiwald, Tim;  Michaud, Véronique;  Rothe, Michael;  Schambach, Axel;  Pfeifer, Richard;  Mason, Chris;  Biasco, Luca;  van Til, Niek P;   - view fewer <#>    (2024)    Preclinical lentiviral vector-mediated hematopoietic stem and progenitor cell gene therapy corrects Pompe disease-related muscle and neurological manifestations.                   Molecular Therapy        10.1016/j.ymthe.2024.09.024 <https://doi.org/10.1016/j.ymthe.2024.09.024>.    (In press).