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...

Descripción completa

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

Internet

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

Ejemplares similares