Regenerative pharmacology in the treatment of genetic diseases: the paradigm of muscular dystrophy

C Mozzetta, G Minetti, PL Puri - The international journal of biochemistry & …, 2009 - Elsevier
Mouse models of dystrophies that show resistance to myostatin blockade should be …
Consistently, ablation of one single allele of p65 could improve muscle pathology and other signs of …

Pharmacologically targeting the primary defect and downstream pathology in Duchenne muscular dystrophy

RJ Fairclough, KJ Perkins, KE Davies - Current gene therapy, 2012 - ingentaconnect.com
… This decreases the efficacy of use in limb girdle muscular … -β/Smad and myostatin signalling
[320]. Whilst heart data was … range of muscular dystrophies [372], the mouse model of LGMD …

The central role of myostatin in skeletal muscle and whole body homeostasis

B Elliott, D Renshaw, S Getting… - Acta …, 2012 - Wiley Online Library
… after 2 weeks cast immobilization significant myostatin mRNA … significantly increases myostatin
expression, where blocking … Finally, in a mouse model of limb-girdle muscular dystrophy, …

Functional and morphological recovery of dystrophic muscles in mice treated with deacetylase inhibitors

GC Minetti, C Colussi, R Adami, C Serra, C Mozzetta… - Nature medicine, 2006 - nature.com
… mice—a model of limb girdle muscular dystrophy (LGMD) 11 … Myostatin blockade at
early stages of the disease provides a … therapy of mouse models of muscular dystrophy is of …

[HTML][HTML] Histone deacetylases: Molecular mechanisms and therapeutic implications for muscular dystrophies

M Sandonà, G Cavioli, A Renzini, A Cedola… - International Journal of …, 2023 - mdpi.com
… ) mouse models, it has been established that HDAC1 and 2 … ameliorate MDs; indeed, myostatin
blockade at early stages of … to milder signs of pathology and an improved function both in …

Myostatin from the heart: local and systemic actions in cardiac failure and muscle wasting

A Breitbart, M Auger-Messier… - … -Heart and …, 2011 - journals.physiology.org
… mediator of skeletal muscle atrophy in mice with heart failure. … NH 2 -terminal prodomain
and the COOH-terminal myostatin … upon myostatin inhibition, indicating that cardiac pathology

Wasting mechanisms in muscular dystrophy

J Shin, MM Tajrishi, Y Ogura, A Kumar - The international journal of …, 2013 - Elsevier
… −/− model of limb-girdle muscular dystrophy 2F (LGMD2F). … The role of myostatin in
pathogenesis of muscular dystrophy … be beneficial in mouse models, it did not restore full function. …

[HTML][HTML] Single SERCA2a therapy ameliorated dilated cardiomyopathy for 18 months in a mouse model of Duchenne muscular dystrophy

NB Wasala, Y Yue, W Lostal, LP Wasala, N Niranjan… - Molecular therapy, 2020 - cell.com
… was indistinguishable from that of normal mice (Figure 2C). … Skeletal muscle histology was
evaluated by hematoxylin and … in a hamster model of limb girdle muscular dystrophy and to …

Muscle wasting diseases: novel targets and treatments

R Furrer, C Handschin - Annual review of pharmacology and …, 2019 - annualreviews.org
… inhibits myostatin (MSTN) expression and thereby contributes to … cancer patients, but it did
not improve functional parameters (74). … dystrophy (DMD or BMD, respectively), or limb-girdle

Myostatin/activin pathway antagonism: molecular basis and therapeutic potential

HQ Han, X Zhou, WE Mitch, AL Goldberg - The international journal of …, 2013 - Elsevier
… Besides these mouse models, cattle, sheep, dogs and a human with a loss… 2). Recent
evidence demonstrates … Based on their key roles in muscle atrophy, the question for therapeutic …