%D 2024
%A John K Yoon
%A Jeffrey W Schindler
%A Mariana Loperfido
%A Cristina Baricordi
%A Mark P DeAndrade
%A Mary E Jacobs
%A Christopher Treleaven
%A Robert N Plasschaert
%A Aimin Yan
%A Cecilia N Barese
%A Yildirim Dogan
%A Vicky Ping Chen
%A Claudia Fiorini
%A Fritz Hull
%A Luigi Barbarossa
%A Zeenath Unnisa
%A Daniel Ivanov
%A Robert H Kutner
%A Swaroopa Guda
%A Christine Oborski
%A Tim Maiwald
%A Véronique Michaud
%A Michael Rothe
%A Axel Schambach
%A Richard Pfeifer
%A Chris Mason
%A Luca Biasco
%A Niek P van Til
%T Preclinical lentiviral vector-mediated hematopoietic stem and progenitor cell gene therapy corrects Pompe disease-related muscle and neurological manifestations
%O This version is the author accepted manuscript. For information on re-use, please refer to the publisher's terms and conditions.
%C United States
%L discovery10197724
%J Molecular Therapy
%I Nature Publishing Group