Scientists at the University of California, Berkeley, have engineered a new way to deliver CRISPR-Cas9 gene-editing technology inside cells and have demonstrated in mice that the technology can repair the mutation that causes ...
Oct 3, 2017
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The ability to cheaply and quickly sequence entire genomes is changing the way diseases are identified and treated. But it is also likely to change the way we make some of the most important and personal decisions of our ...
Aug 2, 2017
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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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Researchers who previously showed that a gene therapy treatment could save the lives of dogs with a deadly disease called myotubular myopathy—a type of muscular dystrophy that affects the skeletal muscles—have found that ...
Jun 8, 2017
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Two papers published today by the Journal of the Royal Society of Medicine, debate gene editing and the health of future generations. Stage and screen actress Kiruna Stamell, who has a rare form of dwarfism, proposes that ...
Apr 27, 2017
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Researchers at The Ohio State University Ross Heart Hospital and Nationwide Children's Hospital have shown early treatment with the heart failure medication eplerenone can improve heart function in young boys with Duchenne ...
Mar 7, 2017
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A new paper, co-written by faculty at Binghamton University, State University of New York, increases the understanding of Duchenne muscular dystrophy (DMD)—one of the most common lethal genetic disorders—and points to ...
Oct 13, 2016
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Muscular dystrophy is a group of rare genetic diseases that cause progressive muscle weakness and deterioration. For decades, researchers have struggled to understand how the disease works and to find suitable treatments.
Oct 4, 2016
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If a disease affects motoneurons, cells that control voluntary muscle activity, researchers should focus their efforts on motoneurons to find potential treatments, right?
Aug 31, 2016
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A new study by a team of scientists from the University of Malta and the Institut de Génétique Moléculaire de Montpellier (CNRS/Université de Montpellier) could help develop treatment strategies for a crippling disorder ...
Aug 1, 2016
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