Safety and preliminary efficacy of LYS-GM101 gene therapy in patients with GM1 gangliosidosis: Results of a phase I/II open-label clinical trial
| Autor: | Ghosh, Arunabha, Héron, Bénédicte, Donald, Aimee, Chang, Harriet, Furness, Laura, Movsesyan, Nina, Ravelli, Claudia, Jones, Simon, Wang, Raymond Y. |
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| Zdroj: | In Molecular Genetics and Metabolism February 2024 141(2) |
| Databáze: | ScienceDirect |
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