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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Results from a clinical trial of eteplirsen, a drug designed to treat Duchenne muscular dystrophy, suggest that the therapy allows participants to walk farther than people treated with placebo and dramatically increases production ...
Aug 8, 2013
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Duchenne muscular dystrophy is a rapidly progressive disease that causes whole-body muscle weakness and atrophy due to deficiency in a protein called dystrophin. Researchers at the University of Missouri, National Center ...
Jul 27, 2017
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In residents of northern latitudes, the content of vitamin D in the body varies naturally throughout the year. This coincides with varying exposure to sunlight, which typically is high in summer and low in winter. Our most ...
Apr 20, 2021
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(HealthDay)—There is a genetic link for strabismus and schizophrenia, with almost half of the genes dysregulated in strabismic medial rectus muscle identified as biomarkers for schizophrenia, according to a study presented ...
May 5, 2015
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Muscular dystrophy describes a group of genetic diseases in which muscles progressively weaken and degenerate, with Duchenne muscular dystrophy (DMD) being the most common. There are no curative treatments for DMD, and available ...
Nov 29, 2021
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Preclinical studies show that gene therapy can improve muscle strength in small- and large-animal models of a fatal congenital pediatric disease known as X-linked myotubular myopathy. The results, appearing in the Jan. 22 ...
Jan 22, 2014
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University of Michigan researchers have discovered a new cause of congenital myopathy: a mutation in a previously uncharacterized gene, according to research published this month in the American Journal of Human Genetics.
Jul 25, 2012
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An experimental gene therapy for Duchenne muscular dystrophy shows promise, a small study suggests.
Jan 28, 2021
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Inhibiting a tiny RNA whose levels significantly increase with age, along with problems like weaker bones and sagging muscles, may be a way to keep our bodies more youthful and healthy, scientists say.
May 2, 2023
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