%0 Journal Article
%A Yoon, John K
%A Schindler, Jeffrey W
%A Loperfido, Mariana
%A Baricordi, Cristina
%A DeAndrade, Mark P
%A Jacobs, Mary E
%A Treleaven, Christopher
%A Plasschaert, Robert N
%A Yan, Aimin
%A Barese, Cecilia N
%A Dogan, Yildirim
%A Chen, Vicky Ping
%A Fiorini, Claudia
%A Hull, Fritz
%A Barbarossa, Luigi
%A Unnisa, Zeenath
%A Ivanov, Daniel
%A Kutner, Robert H
%A Guda, Swaroopa
%A Oborski, Christine
%A Maiwald, Tim
%A Michaud, Véronique
%A Rothe, Michael
%A Schambach, Axel
%A Pfeifer, Richard
%A Mason, Chris
%A Biasco, Luca
%A van Til, Niek P
%D 2024
%F discovery:10197724
%I Nature Publishing Group
%J Molecular Therapy
%T Preclinical lentiviral vector-mediated hematopoietic stem and progenitor cell gene therapy corrects Pompe disease-related muscle and neurological manifestations
%U https://discovery.ucl.ac.uk/id/eprint/10197724/
%Z This version is the author accepted manuscript. For information on re-use, please refer to the publisher's terms and conditions.