Yoon, John K;
Schindler, Jeffrey W;
Loperfido, Mariana;
Baricordi, Cristina;
DeAndrade, Mark P;
Jacobs, Mary E;
Treleaven, Christopher;
... van Til, Niek P; + view all
(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.
(In press).