AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction
| Autor: | Yasuda, Makiko 1, 3, Huston, Marshall W. 2, 3, ∗, Pagant, Silvere 1, Gan, Lin 1, St. Martin, Susan 2, Sproul, Scott 2, Richards, Daniel 2, Ballaron, Stephen 2, Hettini, Khaled 2, Ledeboer, Annemarie 2, Falese, Lillian 2, Cao, Liching 2, Lu, Yanmei 2, Holmes, Michael C. 2, Meyer, Kathleen 2, Desnick, Robert J. 1, Wechsler, Thomas 2 |
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| Zdroj: | In Molecular Therapy - Methods & Clinical Development 11 September 2020 18:607-619 |
| Databáze: | ScienceDirect |
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