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Therapeutic approaches for Duchenne muscular dystrophy
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 …
Eteplirsen in the treatment of Duchenne muscular dystrophy
Duchenne muscular dystrophy is a fatal neuromuscular disorder affecting around one in
3,500–5,000 male births that is characterized by progressive muscular deterioration. It is …
3,500–5,000 male births that is characterized by progressive muscular deterioration. It is …
A high-coverage genome sequence from an archaic Denisovan individual
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 …
coverage (30×) genome sequence of a Denisovan, an extinct relative of Neandertals. The …
Dystrophin protects the sarcolemma from stresses developed during muscle contraction.
BJ Petrof, JB Shrager, HH Stedman, AM Kelly… - Proceedings of the …, 1993 - pnas.org
The protein dystrophin, normally found on the cytoplasmic surface of skeletal muscle cell
membranes, is absent in patients with Duchenne muscular dystrophy as well as mdx (X …
membranes, is absent in patients with Duchenne muscular dystrophy as well as mdx (X …
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 …
A role for the dystrophin-glycoprotein complex as a transmembrane linker between laminin and actin
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 …
the extracellular matrix as well as actin. The 156-kD dystrophin-associated glycoprotein (156 …
Primary structure of dystrophin-associated glycoproteins linking dystrophin to the extracellular matrix
O Ibraghimov-Beskrovnaya, JM Ervasti, CJ Leveille… - Nature, 1992 - nature.com
The primary sequence of two components of the dystrophin–glycoprotein complex has been
established by complementary DNA cloning. The transmembrane 43K and extracellular …
established by complementary DNA cloning. The transmembrane 43K and extracellular …
Molecular physiology of cardiac repolarization
JM Nerbonne, RS Kass - Physiological reviews, 2005 - journals.physiology.org
The heart is a rhythmic electromechanical pump, the functioning of which depends on action
potential generation and propagation, followed by relaxation and a period of refractoriness …
potential generation and propagation, followed by relaxation and a period of refractoriness …
Membrane repair: mechanisms and pathophysiology
ST Cooper, PL McNeil - Physiological reviews, 2015 - journals.physiology.org
Eukaryotic cells have been confronted throughout their evolution with potentially lethal
plasma membrane injuries, including those caused by osmotic stress, by infection from …
plasma membrane injuries, including those caused by osmotic stress, by infection from …
Systemic delivery of full-length dystrophin in Duchenne muscular dystrophy mice
Y Zhou, C Zhang, W **ao, RW Herzog… - Nature Communications, 2024 - nature.com
Current gene therapy for Duchenne muscular dystrophy (DMD) utilizes adeno-associated
virus (AAV) to deliver micro-dystrophin (µDys), which does not provide full protection for …
virus (AAV) to deliver micro-dystrophin (µDys), which does not provide full protection for …