Myostatin/activin pathway antagonism: molecular basis and therapeutic potential

HQ Han, X Zhou, WE Mitch, AL Goldberg - The international journal of …, 2013 - Elsevier
Muscle wasting is associated with a wide range of catabolic diseases. This debilitating loss
of muscle mass and functional capacity reduces the quality of life and increases the risks of …

Pathophysiology of duchenne muscular dystrophy: current hypotheses

N Deconinck, B Dan - Pediatric neurology, 2007 - Elsevier
Duchenne muscular dystrophy is a devastating inherited neuromuscular disorder that affects
one in 3300 live male births. Although the responsible gene and its product, dystrophin …

Large-scale serum protein biomarker discovery in Duchenne muscular dystrophy

Y Hathout, E Brody, PR Clemens… - Proceedings of the …, 2015 - National Acad Sciences
Serum biomarkers in Duchenne muscular dystrophy (DMD) may provide deeper insights
into disease pathogenesis, suggest new therapeutic approaches, serve as acute read-outs …

An antibody blocking activin type II receptors induces strong skeletal muscle hypertrophy and protects from atrophy

E Lach-Trifilieff, GC Minetti, KA Sheppard… - … and cellular biology, 2014 - Am Soc Microbiol
The myostatin/activin type II receptor (ActRII) pathway has been identified to be critical in
regulating skeletal muscle size. Several other ligands, including GDF11 and the activins …

A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy

JR Mendell, Z Sahenk, V Malik, AM Gomez… - Molecular Therapy, 2015 - cell.com
Becker muscular dystrophy (BMD) is a variant of dystrophin deficiency resulting from DMD
gene mutations. Phenotype is variable with loss of ambulation in late teenage or late mid-life …

[HTML][HTML] The role of fibrosis in Duchenne muscular dystrophy

W Klingler, K Jurkat-Rott, F Lehmann-Horn… - Acta …, 2012 - ncbi.nlm.nih.gov
Muscular dystrophies such as Duchenne muscular dystrophy (DMD) are usually
approached as dysfunctions of the affected skeletal myofibres and their force transmission …

[HTML][HTML] Pharmacological inhibition of myostatin suppresses systemic inflammation and muscle atrophy in mice with chronic kidney disease

L Zhang, V Rajan, E Lin, Z Hu, HQ Han, X Zhou… - The FASEB …, 2011 - ncbi.nlm.nih.gov
Chronic kidney disease (CKD) and several other catabolic conditions are characterized by
increased circulating inflammatory cytokines, defects in IGF-1 signaling, abnormal muscle …

Myostatin inhibition in muscle, but not adipose tissue, decreases fat mass and improves insulin sensitivity

T Guo, W Jou, T Chanturiya, J Portas, O Gavrilova… - PloS one, 2009 - journals.plos.org
Myostatin (Mstn) is a secreted growth factor expressed in skeletal muscle and adipose tissue
that negatively regulates skeletal muscle mass. Mstn−/− mice have a dramatic increase in …

Lack of myostatin results in excessive muscle growth but impaired force generation

H Amthor, R Macharia, R Navarrete… - Proceedings of the …, 2007 - National Acad Sciences
The lack of myostatin promotes growth of skeletal muscle, and blockade of its activity has
been proposed as a treatment for various muscle-wasting disorders. Here, we have …

Role of TGF-β signaling in inherited and acquired myopathies

TN Burks, RD Cohn - Skeletal muscle, 2011 - Springer
The transforming growth factor-beta (TGF-β) superfamily consists of a variety of cytokines
expressed in many different cell types including skeletal muscle. Members of this …