Preclinical lentiviral vector-mediated hematopoietic stem and progenitor cell gene therapy corrects Pompe disease-related muscle and neurological manifestations
John K. Yoon, Jeffrey W. Schindler, Mariana Loperfido, Cristina Baricordi, Mark P. DeAndrade, Mary E. Jacobs, Christopher M. Treleaven, Robert N. Plasschaert, Aimin Yan, Cecilia N. Barese, Yildirim Dogan, Vicky Ping Chen, Claudia Fiorini, Fritz Hull, Luigi Barbarossa, , Niek Peter van Til
This study shows lentiviral HSPC gene therapy can reverse Pompe disease symptoms in mice by correcting muscle and neurological issues.
Molecular Therapy · Sep 17, 2024Peer-reviewedNeuroscience