Absence of Dystrophin Disrupts Skeletal Muscle Signaling: Roles of Ca2+, Reactive Oxygen Species, and Nitric Oxide in the Development of Muscular Dystrophy

DG Allen, NP Whitehead… - Physiological …, 2016 - journals.physiology.org
Dystrophin is a long rod-shaped protein that connects the subsarcolemmal cytoskeleton to a
complex of proteins in the surface membrane (dystrophin protein complex, DPC), with further …

Therapeutic approaches for Duchenne muscular dystrophy

TC Roberts, MJA Wood, KE Davies - Nature Reviews Drug Discovery, 2023 - nature.com
Duchenne muscular dystrophy (DMD) is a monogenic muscle-wasting disorder and a
priority candidate for molecular and cellular therapeutics. Although rare, it is the most …

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

CE Nelson, CH Hakim, DG Ousterout, PI Thakore… - Science, 2016 - science.org
Duchenne muscular dystrophy (DMD) is a devastating disease affecting about 1 out of 5000
male births and caused by mutations in the dystrophin gene. Genome editing has the …

The pathogenesis and therapy of muscular dystrophies

S Guiraud, A Aartsma-Rus, NM Vieira… - Annual review of …, 2015 - annualreviews.org
Current molecular genomic approaches to human genetic disorders have led to an
explosion in the identification of the genes and their encoded proteins responsible for these …

Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches

RJ Fairclough, MJ Wood, KE Davies - Nature Reviews Genetics, 2013 - nature.com
Duchenne muscular dystrophy (DMD) is a devastating progressive disease for which there
is currently no effective treatment except palliative therapy. There are several promising …

[HTML][HTML] Muscle and cardiac therapeutic strategies for Duchenne muscular dystrophy: past, present, and future

A Łoboda, J Dulak - Pharmacological Reports, 2020 - Springer
Background Duchenne muscular dystrophy (DMD) is a severe X-linked neuromuscular
childhood disorder that causes progressive muscle weakness and degeneration and results …

[HTML][HTML] Humanizing the mdx mouse model of DMD: the long and the short of it

N Yucel, AC Chang, JW Day, N Rosenthal… - NPJ Regenerative …, 2018 - nature.com
Duchenne muscular dystrophy (DMD) is a common fatal heritable myopathy, with
cardiorespiratory failure occurring by the third decade of life. There is no specific treatment …

[HTML][HTML] Current status of pharmaceutical and genetic therapeutic approaches to treat DMD

C Pichavant, A Aartsma-Rus, PR Clemens, KE Davies… - Molecular Therapy, 2011 - cell.com
Duchenne muscular dystrophy (DMD) is a genetic disease affecting about one in every
3,500 boys. This X-linked pathology is due to the absence of dystrophin in muscle fibers …

Microtubule binding distinguishes dystrophin from utrophin

JJ Belanto, TL Mader, MD Eckhoff… - Proceedings of the …, 2014 - National Acad Sciences
Dystrophin and utrophin are highly similar proteins that both link cortical actin filaments with
a complex of sarcolemmal glycoproteins, yet localize to different subcellular domains within …

Biglycan recruits utrophin to the sarcolemma and counters dystrophic pathology in mdx mice

AR Amenta, A Yilmaz, S Bogdanovich… - Proceedings of the …, 2011 - National Acad Sciences
Duchenne muscular dystrophy (DMD) is caused by mutations in dystrophin and the
subsequent disruption of the dystrophin-associated protein complex (DAPC). Utrophin is a …