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).
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1-s2.0-S1525001624006063-main.pdf - Accepted Version Access restricted to UCL open access staff until 2 April 2025. Download (6MB) |
| Type: | Article |
|---|---|
| Title: | Preclinical lentiviral vector-mediated hematopoietic stem and progenitor cell gene therapy corrects Pompe disease-related muscle and neurological manifestations |
| Location: | United States |
| DOI: | 10.1016/j.ymthe.2024.09.024 |
| Publisher version: | http://dx.doi.org/10.1016/j.ymthe.2024.09.024 |
| Language: | English |
| Additional information: | This version is the author accepted manuscript. For information on re-use, please refer to the publisher's terms and conditions. |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > UCL BEAMS UCL > Provost and Vice Provost Offices > UCL BEAMS > Faculty of Engineering Science > Dept of Biochemical Engineering |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10197724 |
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