Liver-based expression of the human alpha-galactosidase A gene in a murine Fabry model results in continuous therapeutic levels of enzyme activity and effective substrate reduction
| Autor: | Huston, Marshall W. a, Yasuda, Makiko b, Pagant, Silvere b, Martin, Susan St a, Sproul, Scott a, DeKelver, Russell a, Holmes, Michael C. a, Desnick, Robert J. b, Wechsler, Thomas a |
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| Zdroj: | In Molecular Genetics and Metabolism February 2018 123(2):S68-S68 |
| Databáze: | ScienceDirect |
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