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 …

The myostatin gene: physiology and pharmacological relevance

D Joulia-Ekaza, G Cabello - Current opinion in pharmacology, 2007 - Elsevier
Myostatin, which was cloned in 1997, is a potent inhibitor of skeletal muscle growth and
member of the tumour growth factor-β family. Disruption of the myostatin gene in mice …

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 …

Quadrupling muscle mass in mice by targeting TGF-ß signaling pathways

SJ Lee - PloS one, 2007 - journals.plos.org
Myostatin is a transforming growth factor-ß family member that normally acts to limit skeletal
muscle growth. Mice genetically engineered to lack myostatin activity have about twice the …

Muscle diseases: the muscular dystrophies

EM McNally, P Pytel - Annu. Rev. Pathol. Mech. Dis., 2007 - annualreviews.org
Dystrophic muscle disease can occur at any age. Early-or childhood-onset muscular
dystrophies may be associated with profound loss of muscle function, affecting ambulation …

Therapeutic approaches for muscle wasting disorders

GS Lynch, JD Schertzer, JG Ryall - Pharmacology & therapeutics, 2007 - Elsevier
Muscle wasting and weakness are common in many disease states and conditions including
aging, cancer cachexia, sepsis, denervation, disuse, inactivity, burns, HIV-acquired …

Myostatin in the pathophysiology of skeletal muscle

A Bonnieu, G Carnac, B Vernus - Current genomics, 2007 - ingentaconnect.com
Myostatin is an endogenous, negative regulator of muscle growth determining both muscle
fiber number and size. The myostatin pathway is conserved across diverse species ranging …

Muscle growth after postdevelopmental myostatin gene knockout

S Welle, K Bhatt, CA Pinkert, R Tawil… - American Journal of …, 2007 - journals.physiology.org
Constitutive myostatin gene knockout in mice causes excessive muscle growth during
development. To examine the effect of knocking out the myostatin gene after muscle has …

AAV-mediated delivery of a mutated myostatin propeptide ameliorates calpain 3 but not α-sarcoglycan deficiency

M Bartoli, J Poupiot, A Vulin, F Fougerousse, L Arandel… - Gene therapy, 2007 - nature.com
Myostatin is a negative regulator of muscle mass whose inhibition has been proposed as a
therapeutic strategy for muscle-wasting conditions. Indeed, blocking myostatin action …

Overexpression of the cytotoxic T cell (CT) carbohydrate inhibits muscular dystrophy in the dyW mouse model of congenital muscular dystrophy 1A

R Xu, K Chandrasekharan, JH Yoon… - The American journal of …, 2007 - Elsevier
A number of recent studies have demonstrated therapeutic effects of transgenes on the
development of muscle pathology in the mdx mouse model for Duchenne muscular …