Scientists for the first time have used CRISPR gene editing to halt the progression of Duchenne muscular dystrophy (DMD) in a large mammal, according to a study by UT Southwestern that provides a strong indication that a ...
Aug 30, 2018
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A new study from The Ottawa Hospital and the University of Ottawa is poised to completely change our understanding of Duchenne muscular dystrophy and pave the way for far more effective treatments.
Nov 16, 2015
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A new multi-institution study spearheaded by researchers at Florida State University and the University of California, Los Angeles suggests a tiny protein could play a major role in combating heart failure related to Duchenne ...
Jun 14, 2019
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Using a novel genetic 'editing' technique, Duke University biomedical engineers have been able to repair a defect responsible for one of the most common inherited disorders, Duchenne muscular dystrophy, in cell samples from ...
Jun 4, 2013
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A team of researchers from the U.S. and Germany describes a novel CRISPR approach to produce healthy heart muscle using pluripotent stem cells from Duchenne muscular dystrophy (DMD) patients. In their paper published on the ...
A protein that has shown early promise in preventing the loss of muscle function in mouse models of Duchenne muscular dystrophy, has been found in a new study to be a key player in the process of joining nerves to muscles.
Feb 14, 2012
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(Medical Xpress) -- A team led by scientists at UCL, funded by the Medical Research Council (MRC) and AVI BioPharma, have made an important breakthrough in the development of a treatment for Duchenne Muscular Dystrophy (DMD).
Jul 25, 2011
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An experimental drug designed to fit a DNA patch in a flawed gene has cleared an important hurdle in tests on boys struck by a tragic muscle-wasting disease, a British study on Monday says.
Jul 25, 2011
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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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Researchers at the University of Minnesota's Lillehei Heart Institute have combined genetic repair with cellular reprogramming to generate stem cells capable of muscle regeneration in a mouse model for Duchenne Muscular Dystrophy ...
Mar 5, 2013
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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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