Silencing Nfix rescues muscular dystrophy by delaying muscle regeneration.
Nat Commun
; 8(1): 1055, 2017 10 20.
Article
em En
| MEDLINE
| ID: mdl-29057908
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.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Regeneração
/
Fatores de Transcrição NFI
/
Músculos
/
Distrofias Musculares
Limite:
Animals
Idioma:
En
Revista:
Nat Commun
Ano de publicação:
2017
Tipo de documento:
Article