Full-length ATP7B reconstituted through protein trans-splicing corrects Wilson disease in mice.
Padula A, Petruzzelli R, Philbert SA, Church SJ, Esposito F, Campione S, Monti M, Capolongo F, Perna C, Nusco E, Schmidt HH, Auricchio A, Cooper GJS, Polishchuk R, Piccolo P.
Padula A, et al. Among authors: cooper gjs.
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Mol Ther Methods Clin Dev. 2022.
PMID: 36092366
Free PMC article.