eprintid: 10197724
rev_number: 7
eprint_status: archive
userid: 699
dir: disk0/10/19/77/24
datestamp: 2024-09-30 12:37:15
lastmod: 2024-09-30 12:37:15
status_changed: 2024-09-30 12:37:15
type: article
metadata_visibility: show
sword_depositor: 699
creators_name: Yoon, John K
creators_name: Schindler, Jeffrey W
creators_name: Loperfido, Mariana
creators_name: Baricordi, Cristina
creators_name: DeAndrade, Mark P
creators_name: Jacobs, Mary E
creators_name: Treleaven, Christopher
creators_name: Plasschaert, Robert N
creators_name: Yan, Aimin
creators_name: Barese, Cecilia N
creators_name: Dogan, Yildirim
creators_name: Chen, Vicky Ping
creators_name: Fiorini, Claudia
creators_name: Hull, Fritz
creators_name: Barbarossa, Luigi
creators_name: Unnisa, Zeenath
creators_name: Ivanov, Daniel
creators_name: Kutner, Robert H
creators_name: Guda, Swaroopa
creators_name: Oborski, Christine
creators_name: Maiwald, Tim
creators_name: Michaud, Véronique
creators_name: Rothe, Michael
creators_name: Schambach, Axel
creators_name: Pfeifer, Richard
creators_name: Mason, Chris
creators_name: Biasco, Luca
creators_name: van Til, Niek P
title: Preclinical lentiviral vector-mediated hematopoietic stem and progenitor cell gene therapy corrects Pompe disease-related muscle and neurological manifestations
ispublished: inpress
divisions: UCL
divisions: B04
divisions: F47
note: This version is the author accepted manuscript. For information on re-use, please refer to the publisher's terms and conditions.
date: 2024-09-17
date_type: published
publisher: Nature Publishing Group
official_url: http://dx.doi.org/10.1016/j.ymthe.2024.09.024
full_text_type: other
language: eng
verified: verified_manual
elements_id: 2322394
doi: 10.1016/j.ymthe.2024.09.024
medium: Print-Electronic
pii: S1525-0016(24)00606-3
lyricists_name: Mason, Christopher
lyricists_id: CMASO20
actors_name: Flynn, Bernadette
actors_id: BFFLY94
actors_role: owner
full_text_status: restricted
publication: Molecular Therapy
event_location: United States
citation:        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).   
 
document_url: https://discovery.ucl.ac.uk/id/eprint/10197724/1/1-s2.0-S1525001624006063-main.pdf