Full-length ATP7B reconstituted through protein trans-splicing corrects Wilson disease in mice
| Autor: | Padula, Agnese, Petruzzelli, Raffaella, Philbert, Sasha A., Church, Stephanie J., Esposito, Federica, Campione, Severo, Monti, Marcello, Capolongo, Filomena, Perna, Claudia, Nusco, Edoardo, Schmidt, Hartmut H., Auricchio, Alberto, Cooper, Garth J.S., Polishchuk, Roman, Piccolo, Pasquale |
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| Zdroj: | In Molecular Therapy - Methods & Clinical Development 8 September 2022 26:495-504 |
| Databáze: | ScienceDirect |
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