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 …
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 …
[HTML][HTML] The Dystrophin Complex: structure, function and implications for therapy
Q Gao, EM McNally - Comprehensive physiology, 2015 - ncbi.nlm.nih.gov
The dystrophin complex stabilizes the plasma membrane of striated muscle cells. Loss of
function mutations in the genes encoding dystrophin, or the associated proteins, triggers …
function mutations in the genes encoding dystrophin, or the associated proteins, triggers …
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 …
by loss-of-function mutations in the dystrophin gene. Currently, there is no cure. A highly …
Emerging therapies for Duchenne muscular dystrophy
Duchenne muscular dystrophy is an X-linked disease caused by the absence of functional
dystrophin in the muscle cells. Major advances have led to the development of gene …
dystrophin in the muscle cells. Major advances have led to the development of gene …
Drug development progress in duchenne muscular dystrophy
J Deng, J Zhang, K Shi, Z Liu - Frontiers in Pharmacology, 2022 - frontiersin.org
Duchenne muscular dystrophy (DMD) is a severe, progressive, and incurable X-linked
disorder caused by mutations in the dystrophin gene. Patients with DMD have an absence of …
disorder caused by mutations in the dystrophin gene. Patients with DMD have an absence of …
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 …
Duchenne muscular dystrophy: Disease mechanism and therapeutic strategies
A Bez Batti Angulski, N Hosny, H Cohen… - Frontiers in …, 2023 - frontiersin.org
Duchenne muscular dystrophy (DMD) is a severe, progressive, and ultimately fatal disease
of skeletal muscle wasting, respiratory insufficiency, and cardiomyopathy. The identification …
of skeletal muscle wasting, respiratory insufficiency, and cardiomyopathy. The identification …
Function and genetics of dystrophin and dystrophin-related proteins in muscle
DJ Blake, A Weir, SE Newey… - Physiological …, 2002 - journals.physiology.org
The X-linked muscle-wasting disease Duchenne muscular dystrophy is caused by mutations
in the gene encoding dystrophin. There is currently no effective treatment for the disease; …
in the gene encoding dystrophin. There is currently no effective treatment for the disease; …
Muscle spindle function in healthy and diseased muscle
S Kröger, B Watkins - Skeletal Muscle, 2021 - Springer
Almost every muscle contains muscle spindles. These delicate sensory receptors inform the
central nervous system (CNS) about changes in the length of individual muscles and the …
central nervous system (CNS) about changes in the length of individual muscles and the …