TY  - INPR
AV  - restricted
TI  - Preclinical lentiviral vector-mediated hematopoietic stem and progenitor cell gene therapy corrects Pompe disease-related muscle and neurological manifestations
Y1  - 2024/09/17/
ID  - discovery10197724
UR  - http://dx.doi.org/10.1016/j.ymthe.2024.09.024
N1  - This version is the author accepted manuscript. For information on re-use, please refer to the publisher's terms and conditions.
PB  - Nature Publishing Group
JF  - Molecular Therapy
A1  - Yoon, John K
A1  - Schindler, Jeffrey W
A1  - Loperfido, Mariana
A1  - Baricordi, Cristina
A1  - DeAndrade, Mark P
A1  - Jacobs, Mary E
A1  - Treleaven, Christopher
A1  - Plasschaert, Robert N
A1  - Yan, Aimin
A1  - Barese, Cecilia N
A1  - Dogan, Yildirim
A1  - Chen, Vicky Ping
A1  - Fiorini, Claudia
A1  - Hull, Fritz
A1  - Barbarossa, Luigi
A1  - Unnisa, Zeenath
A1  - Ivanov, Daniel
A1  - Kutner, Robert H
A1  - Guda, Swaroopa
A1  - Oborski, Christine
A1  - Maiwald, Tim
A1  - Michaud, Véronique
A1  - Rothe, Michael
A1  - Schambach, Axel
A1  - Pfeifer, Richard
A1  - Mason, Chris
A1  - Biasco, Luca
A1  - van Til, Niek P
ER  -