Silencing Nfix rescues muscular dystrophy by delaying muscle regeneration

Giuliana Rossi, Chiara Bonfanti, Stefania Antonini, Mattia Bastoni, Stefania Monteverde, Anna Innocenzi, Marielle Saclier, Valentina Taglietti and Graziella Messina ()
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Giuliana Rossi: University of Milan
Chiara Bonfanti: University of Milan
Stefania Antonini: University of Milan
Mattia Bastoni: University of Milan
Stefania Monteverde: University of Milan
Anna Innocenzi: San Raffaele Scientific Institute
Marielle Saclier: University of Milan
Valentina Taglietti: University of Milan
Graziella Messina: University of Milan

Nature Communications, 2017, vol. 8, issue 1, 1-12

Abstract: Abstract Muscular dystrophies are severe disorders due to mutations in structural genes, and are characterized by skeletal muscle wasting, compromised patient mobility, and respiratory functions. Although previous works suggested enhancing regeneration and muscle mass as therapeutic strategies, these led to no long-term benefits in humans. Mice lacking the transcription factor Nfix have delayed regeneration and a shift toward an oxidative fiber type. Here, we show that ablating or silencing the transcription factor Nfix ameliorates pathology in several forms of muscular dystrophy. Silencing Nfix in postnatal dystrophic mice, when the first signs of the disease already occurred, rescues the pathology and, conversely, Nfix overexpression in dystrophic muscles increases regeneration and markedly exacerbates the pathology. We therefore offer a proof of principle for a novel therapeutic approach for muscular dystrophies based on delaying muscle regeneration.

Date: 2017
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DOI: 10.1038/s41467-017-01098-y

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