Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy

JW McGreevy, CH Hakim… - Disease models & …, 2015 - journals.biologists.com
Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disorder. It is caused
by loss-of-function mutations in the dystrophin gene. Currently, there is no cure. A highly …

Extracellular matrix: the driving force of mammalian diseases

RV Iozzo, MA Gubbiotti - Matrix Biology, 2018 - Elsevier
Like the major theme of a Mozart concerto, the immense and pervasive extracellular matrix
drives each movement and ultimately closes the symphony, embracing a unique role as the …

Zebrafish: an emerging model system for human disease and drug discovery

G Kari, U Rodeck, AP Dicker - Clinical Pharmacology & …, 2007 - Wiley Online Library
In vivo studies represent an essential step in drug development and currently rely largely on
mice, yet limitations of mammalian models motivated the search for complementary …

[HTML][HTML] Efficient and Reproducible Myogenic Differentiation from Human iPS Cells: Prospects for Modeling Miyoshi Myopathy In Vitro

A Tanaka, K Woltjen, K Miyake, A Hotta, M Ikeya… - PloS one, 2013 - journals.plos.org
The establishment of human induced pluripotent stem cells (hiPSCs) has enabled the
production of in vitro, patient-specific cell models of human disease. In vitro recreation of …

[HTML][HTML] MicroRNA-486–dependent modulation of DOCK3/PTEN/AKT signaling pathways improves muscular dystrophy–associated symptoms

MS Alexander, JC Casar, N Motohashi… - The Journal of …, 2014 - Am Soc Clin Investig
Duchenne muscular dystrophy (DMD) is caused by mutations in the gene encoding
dystrophin, which results in dysfunctional signaling pathways within muscle. Previously, we …

miRNAS in normal and diseased skeletal muscle

I Eisenberg, MS Alexander… - Journal of cellular and …, 2009 - Wiley Online Library
• Introduction• Mechanisms of miRNA‐mediated repression• miRNA regulatory network in
muscle‐Muscle‐specific miRNAs‐Non‐muscle‐specific miRNAs• miRNA signatures as …

A central function for perlecan in skeletal muscle and cardiovascular development

JJ Zoeller, A McQuillan, J Whitelock, SY Ho… - The Journal of cell …, 2008 - rupress.org
Perlecan's developmental functions are difficult to dissect in placental animals because
perlecan disruption is embryonic lethal. In contrast to mammals, cardiovascular function is …

Zebrafish relatively relaxed mutants have a ryanodine receptor defect, show slow swimming and provide a model of multi-minicore disease

H Hirata, T Watanabe, J Hatakeyama, SM Sprague… - 2007 - journals.biologists.com
Wild-type zebrafish embryos swim away in response to tactile stimulation. By contrast,
relatively relaxed mutants swim slowly due to weak contractions of trunk muscles …

[HTML][HTML] Model Organisms in the Fight against Muscular Dystrophy: Lessons from Drosophila and Zebrafish

E Plantié, M Migocka-Patrzałek, M Daczewska, K Jagla - Molecules, 2015 - mdpi.com
Muscular dystrophies (MD) are a heterogeneous group of genetic disorders that cause
muscle weakness, abnormal contractions and muscle wasting, often leading to premature …

Cell-lineage regulated myogenesis for dystrophin replacement: a novel therapeutic approach for treatment of muscular dystrophy

E Kimura, JJ Han, S Li, B Fall, J Ra… - Human molecular …, 2008 - academic.oup.com
Duchenne muscular dystrophy (DMD) is characterized in skeletal muscle by cycles of
myofiber necrosis and regeneration leading to loss of muscle fibers and replacement with …