Duchenne muscular dystrophy (DMD) is a muscle degeneration disorder caused by mutations affecting the dystrophin gene. On August 24 in the journal Stem Cell Reports, researchers show how a dual CRISPR RNA method restored ...
Aug 24, 2023
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A group of Ludwig Maximilian University scientists led by Eckhard Wolf has developed a porcine DMD model with a mutation that mimics the hallmarks of the human disease but develops them in an accelerated mode. The study is ...
Jul 13, 2023
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A cell therapy developed by the executive director of the Smidt Heart Institute stabilizes weakened muscles—including the heart muscle—in Duchenne muscular dystrophy patients, a new study published in the international ...
Apr 13, 2022
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A novel gene editing technique developed by researchers at The Ohio State University Wexner Medical Center and College of Medicine is showing promise in correcting genetic mutations in Duchenne muscular dystrophy (DMD) and ...
Jun 25, 2021
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UT Southwestern scientists successfully employed a new type of gene therapy to treat mice with Duchenne muscular dystrophy (DMD), uniquely utilizing CRISPR-Cas9-based tools to restore a large section of the dystrophin protein ...
May 3, 2021
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Duchenne muscular dystrophy (DMD) is the most common muscle disease in children and is passed on by X-linked recessive inheritance. Characteristic is a progressive muscular atrophy. The disease often results in death before ...
Aug 27, 2020
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Academics at Royal Holloway have developed a new genetic medicine that has been given the go ahead by the U.S Food and Drug Association (FDA) to be prescribed globally to a subset of patients with Duchenne muscular dystrophy ...
Feb 25, 2020
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Researchers identified a group of small molecules that may open the door to developing new therapies for Duchenne muscular dystrophy (DMD), an as-yet-uncured disease that results in devastating muscle weakening and loss. ...
Feb 24, 2020
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Duchenne type muscular dystrophy (DMD) is the most common hereditary muscular disease among children, leaving them wheelchair-bound before the age of 12 and reducing life expectancy. Researchers at Technical University of ...
Jan 28, 2020
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A new therapeutic being tested by University of Alberta researchers is showing early promise as a more effective treatment that could help nearly half of patients with Duchenne muscular dystrophy (DMD).
Sep 25, 2019
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Dystrophin is a rod-shaped cytoplasmic protein, and a vital part of a protein complex that connects the cytoskeleton of a muscle fiber to the surrounding extracellular matrix through the cell membrane. This complex is variously known as the costamere or the dystrophin-associated protein complex. Many muscle proteins, such as α-dystrobrevin, syncoilin, synemin, sarcoglycan, dystroglycan, and sarcospan, colocalize with dystrophin at the costamere.
The Dystrophin gene is the longest human gene known on DNA level, covering 2.4 megabases (0.08% of the human genome) at locus Xp21. However, it does not encode the longest protein known in humans, which is titin. The primary transcript measures about 2,400 kilobases and takes 16 hours to transcribe; the mature mRNA measures 14.0 kilobases. The 79 exons code for a protein of over 3500 amino acid residues.
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