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

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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Bibliographic Details
Main Authors: Agnese Padula, Raffaella Petruzzelli, Sasha A. Philbert, Stephanie J. Church, Federica Esposito, Severo Campione, Marcello Monti, Filomena Capolongo, Claudia Perna, Edoardo Nusco, Hartmut H. Schmidt, Alberto Auricchio, Garth J.S. Cooper, Roman Polishchuk, Pasquale Piccolo
Format: Article
Language:English
Published: Elsevier 2022-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
adeno-associated vectors
split inteins
protein trans-splicing
Wilson disease
copper storage
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050122001140

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http://www.sciencedirect.com/science/article/pii/S2329050122001140

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