Duchenne muscular dystrophy trajectory in R-DMDdel52 preclinical rat model identifies COMP as biomarker of fibrosis

Duchenne muscular dystrophy trajectory in R-DMDdel52 preclinical rat model identifies COMP as biomarker of fibrosis

Duchenne muscular dystrophy (DMD) is a fatal muscle-wasting disorder caused by mutations in the Dystrophin gene and for which there is currently no cure. To bridge the gap between preclinical and therapeutic evaluation studies, we have generated a rat model for DMD that carries an exon 52 deletion (...

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Bibliographic Details
Main Authors: Authier, F.-J (Author), Bronisz-Budzyńska, I. (Author), Cardone, N. (Author), Coulpier, F. (Author), Gentil, C. (Author), Goddard, M. (Author), Kefi, K. (Author), Lafuste, P. (Author), Malfatti, E. (Author), Mirciloglu, B. (Author), Periou, B. (Author), Pietri-Rouxel, F. (Author), Relaix, F. (Author), Rodrigues, M. (Author), Taglietti, V. (Author), Tiret, L. (Author)
Format: Article
Language:English
Published: BioMed Central Ltd 2022
Subjects:
Duchenne muscular dystrophy
Long QT
Preclinical modelling
scRNAseq
Skeletal muscle
Translational medicine
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