by Mary Ann Liebert, Inc Fibrotic remodeling in skeletal muscle and heart of DMDMDX rats and impact of delandistrogene moxeparvovec treatment. A recent study published in the journal Human Gene Therapy evaluated the long-term survival and cardiac efficacy of the gene therapy delandistrogene moxeparvovec in a rat model of Duchenne muscular dystrophy (DMD). Delandistrogene moxeparvovec uses an adeno-associated viral (AAV) vector...
Tag: <span>muscular dystrophy</span>
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Mouse model reveals liver involvement in muscular dystrophy
by Liz Ahlberg Touchstone, University of Illinois at Urbana-Champaign In muscular dystrophy type 1, mutated DNA begets toxic RNA that alter liver function, including susceptibility to fatty liver disease and hypersensitivity to drugs. Understanding these effects is crucial for developing treatments, as many have proven toxic in patients. Credit: Haneni Bae, University of Illinois at Urbana-Champaign...
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Scrutinizing cells for clues to a treatment for muscular dystrophy
Characteristics of Ad-MSCs, BM-MSCs, and XF-iMSCs. Credit: Stem Cell Research & Therapy (2024). DOI: 10.1186/s13287-024-03951-6 A research team led by Associate Professor Hidetoshi Sakurai and Researcher Nana Takenaka-Ninagawa recently demonstrated the superior therapeutic potential of iPS cell-derived mesenchymal stromal cells (iMSCs) compared to primary MSCs as a potential treatment for Ullrich congenital muscular dystrophy. The study is published in Stem...
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New gene therapy approach shows promise for Duchenne muscular dystrophy
JULY 25, 2024 by Jackie Maupin, Indiana University School of Medicine Design and test of split intein constructs to assemble FL-dystrophin. Credit: Nature Communications (2024). DOI: 10.1038/s41467-024-50569-6Indiana University School of Medicine researchers have made a significant breakthrough in developing a new gene therapy approach that restores full-length dystrophin protein, which could lead to new treatments...
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Duchenne Muscular Dystrophy Gene Therapy Safe, Effective at 4 Years
Nancy A. Melville November 06, 2023 PHOENIX — Children with Duchenne muscular dystrophy (DMD) treated with the only gene therapy to date to be approved for treatment of disease in the United States show sustained maintenance of motor function after 4 years, compared with untreated patients who showed significant decline over the same time period, new research...
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Researchers target protein that can slow down muscular dystrophy
by Laurie Fickman, University of Houston Graphical abstract. Credit: JCI Insight (2023). DOI: 10.1172/jci.insight.164768 A team of researchers at the University of Houston College of Pharmacy is reporting that by manipulating TAK1, a signaling protein that plays an important role in development of the immune system, they can slow down disease progression and improve muscle function in Duchenne muscular dystrophy...
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Could a novel small molecule slow or reverse the effects of Duchenne muscular dystrophy?
by Wiley Histopathology of gastrocnemius muscle from patient who died of pseudohypertrophic muscular dystrophy, Duchenne type. Cross section of muscle shows extensive replacement of muscle fibers by adipose cells. Credit: Wikimedia Commons/Public Domain In a new study published in The FASEB Journal, investigators demonstrated the potential of a molecule that may help overcome some of the...
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Toxic protein linked to muscular dystrophy and arhinia
by National Institutes of Health Credit: Pixabay/CC0 Public Domain Researchers at the National Institutes of Health and their colleagues have found that a toxic protein made by the body called DUX4 may be the cause of two very different rare genetic disorders. For patients who have facioscapulohumeral muscular dystrophy (FSHD), or a rare facial malformation called arhinia,...
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Cancer drug shows potential as treatment for muscular dystrophy
by University of British Columbia Effects of CSF1R inhibitors – and the absence of self-renewing resident macrophages – on muscle regeneration in mice. Credit: F. Babaeijandaghi, et al., Science Translational Medicine (2022) Researchers at the University of British Columbia’s School of Biomedical Engineering have discovered that an existing cancer drug could have potential as a treatment for muscular...
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Study reveals how Duchenne muscular dystrophy causes heart rhythm problems
ELIFE IMAGE: STEM CELL-DERIVED CARDIOMYOCYTES PRODUCED TO STUDY ARRHYTHMIC MECHANISMS CREDIT: FÁTIMA LOIS The results help explain why as many as 60% of patients with DMD have potentially life-threatening heart rhythm abnormalities. They may also suggest potential treatment strategies for heart problems in people with DMD. Mutations in a gene that encodes a muscle-protecting protein...