Category Ranking

98%

Total Visits

921

Avg Visit Duration

2 minutes

Citations

20

Article Abstract

Duchenne Muscular Dystrophy is a devastating disease caused by the absence of a functional rod-shaped cytoplasmic protein called dystrophin. Several avenues are being developed aimed to restore dystrophin expression in boys affected by this X-linked disease. However, its complete cure is likely to need combinational approaches which may include regimes aimed at restoring muscle mass. Augmenting muscle growth through the manipulation of the Myostatin/Activin signalling axis has received much attention. However, we have recently shown that while manipulation of this axis in wild type mice using the sActRIIB ligand trap indeed results in muscle growth, it also had a detrimental impact on the testis. Here we examined the impact of administering a powerful Myostatin/Activin antagonist in two mouse models of Duchenne Muscular Dystrophy. We report that whilst the impact on muscle growth was not always positive, both models showed attenuated testis development. Sperm number, motility and ultrastructure were significantly affected by the sActRIIB treatment. Our report suggests that interventions based on Myostatin/Activin should investigate off-target effects on tissues as well as muscle.

Download full-text PDF

Source
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7385695PMC
http://dx.doi.org/10.4081/ejtm.2019.8904DOI Listing

Publication Analysis

Top Keywords

duchenne muscular
12
muscular dystrophy
12
muscle growth
12
mouse models
8
models duchenne
8
muscle
6
diminution sperm
4
sperm quantity
4
quantity quality
4
quality mouse
4

Similar Publications

Background: Heart transplant (HTx) in dystrophy patients has been shown to have a similar survival to cardiomyopathy from other causes, but postoperative rehabilitation remains an issue. This study aimed to review and analyze the reports in the literature to determine whether pre- and post-transplant functional status along with wheelchair dependence in dystrophy patients can influence post-HTx outcomes.

Research Design And Methods: Relevant databases were queried for all case reports and case series regarding HTx in patients with dystrophy-associated cardiomyopathy published in the literature.

View Article and Find Full Text PDF

Fighting for every beat: cardiac therapies in Duchenne muscular dystrophy.

Skelet Muscle

September 2025

Sorbonne Université, Inserm, Institut de Myologie, Centre de Recherche en Myologie, Paris, France.

Duchenne muscular dystrophy (DMD) is a severe, progressive genetic disorder caused by mutations in the DMD gene, resulting in the absence of dystrophin-a key structural protein at the sarcolemma. As the disease progresses, cardiac involvement becomes a leading cause of morbidity and mortality. By adolescence or early adulthood, many patients develop dilated cardiomyopathy and arrhythmias.

View Article and Find Full Text PDF

The goal of this paper is to estimate an optimal combination of biomarkers for individuals with Duchenne muscular dystrophy (DMD), which provides the most sensitive combinations of biomarkers to assess disease progression (in this case, optimal with respect to standardized response mean (SRM) for 4 muscle biomarkers). The biomarker data is incomplete (missing and irregular) multivariate longitudinal data. We propose a normal model with structured covariance designed for our setting.

View Article and Find Full Text PDF

Duchenne muscular dystrophy (DMD) is the most common inherited muscle disease. There are currently few effective therapies to treat the disease, although many approaches are being pursued. Certain histone deacetylase inhibitors (HDACi) have been shown to ameliorate DMD phenotypes in mouse and zebrafish models, and the HDACi givinostat has recently gained FDA approval for DMD.

View Article and Find Full Text PDF