First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B
| Autor: | Harmatz, Paul, Prada, Carlos E., Burton, Barbara K., Lau, Heather, Kessler, Craig M., Cao, Liching, Falaleeva, Marina, Villegas, Andres G., Zeitler, Jennifer, Meyer, Kathleen, Miller, Weston, Wong Po Foo, Cheryl, Vaidya, Sagar, Swenson, Wendy, Shiue, Lisa H., Rouy, Didier, Muenzer, Joseph |
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| Zdroj: | In Molecular Therapy 7 December 2022 30(12):3587-3600 |
| Databáze: | ScienceDirect |
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