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 …
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 …
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 …
male births and caused by mutations in the dystrophin gene. Genome editing has the …
The pathogenesis and therapy of muscular dystrophies
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 …
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 …
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 …
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 …
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 …
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 …
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 …
subsequent disruption of the dystrophin-associated protein complex (DAPC). Utrophin is a …