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., Yasuda, Makiko, Pagant, Silvere, Martin, Susan St, Sproul, Scott, DeKelver, Russell, Holmes, Michael C., Desnick, Robert J., Wechsler, Thomas |
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| Zdroj: | In Molecular Genetics and Metabolism February 2018 123(2):S68-S68 |
| Databáze: | ScienceDirect |
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