Hematopoietic stem cell transplantation leads to biochemical and functional correction in two mouse models of acid ceramidase deficiency
| Autor: | Rybova, Jitka, Sundararajan, Teresa, Kuchar, Ladislav, Dlugi, Theresa A., Ruzicka, Petr, McKillop, William M., Medin, Jeffrey A. |
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| Zdroj: | In Molecular Therapy 2 October 2024 32(10):3402-3421 |
| Databáze: | ScienceDirect |
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