Progressively shortening telomeres—the protective caps on the end of chromosomes—may be responsible for the weakened, enlarged hearts that kill many sufferers of Duchenne muscular dystrophy, according to a study by researchers ...
Oct 31, 2016
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For the first time, researchers have employed a gene-editing technique involving low-dose irradiation to repair patient cells, according to a study published in the journal Stem Cells Translational Medicine. This method, ...
Jul 22, 2015
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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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Spinal muscular atrophy, or SMA, is the leading genetic cause of infant death. Less than a decade ago, Cold Spring Harbor Laboratory (CSHL) Professor Adrian Krainer showed this brutal disease can be treated by tweaking a ...
Mar 6, 2024
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In the past few months significant insights into the molecular controls of damaged tissue and how to manipulate them for better regeneration has been revealed for treating rare muscular disorders, which also extends to the ...
Oct 22, 2014
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The advent of therapeutic interventions for spinal muscular atrophy (SMA) has increased the importance of presymptomatic diagnosis and treatment. When to start treatment in children with less severe disease remains controversial. ...
Jun 11, 2020
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Researchers at the Center for iPS Cell Research and Application (CiRA), Kyoto University, show that induced pluripotent stem (iPS) cells can be used to correct genetic mutations that cause Duchenne muscular dystrophy (DMD). ...
Nov 26, 2014
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Britain's fertility regulator says it has found broad public support for in vitro fertilization techniques that allow babies to be created with DNA from three people for couples at risk of passing on potentially fatal genetic ...
Mar 20, 2013
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Mutations of the gene Lmna previously thought to be directly responsible for a group of laminopathies—serious developmental conditions including premature aging and a form of muscular dystrophy—in fact cause them by allowing ...
Aug 29, 2012
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NYU biologists have created model neurons with greater precision and efficiency than have been achieved in the past. Their breakthrough, which appeared this fall in a pair of papers in the journal Nature Neuroscience, has ...
Dec 12, 2013
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