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Full-length ATP7B reconstituted through protein trans-splicing corrects Wilson disease in mice

Wilson disease (WD) is a genetic disorder of copper homeostasis, caused by deficiency of the copper transporter ATP7B. Gene therapy with recombinant adeno-associated vectors (AAV) holds promises for WD treatment. However, the full-length human ATP7B gene exceeds the limited AAV cargo capacity, hampe...

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Detalles Bibliográficos
Autores principales:	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
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2022
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9436707/ https://www.ncbi.nlm.nih.gov/pubmed/36092366 http://dx.doi.org/10.1016/j.omtm.2022.08.004

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