CFTR corrector C17 is effective in muscular dystrophy, in vivo proof of concept in LGMDR3.

Autor: Scano M; Department of Biomedical Sciences, University of Padova, Padova, Italy., Benetollo A; Department of Biomedical Sciences, University of Padova, Padova, Italy., Nogara L; Venetian Institute of Molecular Medicine, University of Padova, Padova, Italy., Bondì M; Department of Biomedical Sciences, University of Padova, Padova, Italy., Dalla Barba F; Department of Biomedical Sciences, University of Padova, Padova, Italy., Soardi M; Department of Biomedical Sciences, University of Padova, Padova, Italy., Furlan S; Neuroscience Institute - Italian National Research Council (CNR), Padova, Italy., Akyurek EE; Department of Comparative Biomedicine and Food Science, University of Padova, Padova, Italy., Caccin P; Department of Biomedical Sciences, University of Padova, Padova, Italy., Carotti M; Department of Biomedical Sciences, University of Padova, Padova, Italy., Sacchetto R; Department of Comparative Biomedicine and Food Science, University of Padova, Padova, Italy., Blaauw B; Department of Biomedical Sciences, University of Padova, Padova, Italy.; Venetian Institute of Molecular Medicine, University of Padova, Padova, Italy., Sandonà D; Department of Biomedical Sciences, University of Padova, Padova, Italy.

Publikováno v: Human molecular genetics [Hum Mol Genet] 2022 Feb 21; Vol. 31 (4), pp. 499-509.