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 …

Extracellular matrix: an important regulator of cell functions and skeletal muscle development

W Zhang, Y Liu, H Zhang - Cell & bioscience, 2021 - Springer
Extracellular matrix (ECM) is a kind of connective tissue in the cell microenvironment, which
is of great significance to tissue development. ECM in muscle fiber niche consists of three …

A high-coverage genome sequence from an archaic Denisovan individual

M Meyer, M Kircher, MT Gansauge, H Li, F Racimo… - Science, 2012 - science.org
We present a DNA library preparation method that has allowed us to reconstruct a high-
coverage (30×) genome sequence of a Denisovan, an extinct relative of Neandertals. The …

A role for the dystrophin-glycoprotein complex as a transmembrane linker between laminin and actin

JM Ervasti, KP Campbell - The Journal of cell biology, 1993 - rupress.org
The dystrophin-glycoprotein complex was tested for interaction with several components of
the extracellular matrix as well as actin. The 156-kD dystrophin-associated glycoprotein (156 …

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; …

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 …

Nitric oxide synthase complexed with dystrophin and absent from skeletal muscle sarcolemma in Duchenne muscular dystrophy

JE Brenman, DS Chao, H **a, K Aldape, DS Bredt - Cell, 1995 - cell.com
Nitric oxide (NO) is synthesized in skeletal muscle by neuronal-type NO synthase (nNOS),
which is localized to sarcolemma of fast-twitch fibers. Synthesis of NO in active muscle …

[HTML][HTML] Utrophin-dystrophin-deficient mice as a model for Duchenne muscular dystrophy

AE Deconinck, JA Rafael, JA Skinner, SC Brown… - Cell, 1997 - cell.com
The absence of dystrophin at the muscle membrane leads to Duchenne muscular dystrophy
(DMD), a severe muscle-wasting disease that is inevitably fatal in early adulthood. In …

Overview of the muscle cytoskeleton

CA Henderson, CG Gomez, SM Novak… - Comprehensive …, 2017 - pmc.ncbi.nlm.nih.gov
Cardiac and skeletal striated muscles are intricately designed machines responsible for
muscle contraction. Coordination of the basic contractile unit, the sarcomere, and the …

Skeletal and cardiac myopathies in mice lacking utrophin and dystrophin: a model for Duchenne muscular dystrophy

RM Grady, H Teng, MC Nichol, JC Cunningham… - Cell, 1997 - cell.com
Dystrophin is a cytoskeletal protein of muscle fibers; its loss in humans leads to Duchenne
muscular dystrophy, an inevitably fatal wasting of skeletal and cardiac muscle. mdx mice …