A multi-institutional research group has succeeded in developing an exon-skipping therapy using nucleic-acid therapeutics for Alport syndrome, an incurable kidney disease that can progress to renal failure. Disease model ...
Jun 25, 2020
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Johns Hopkins researchers have identified a natural mechanism that might one day be used to block the expression of the mutated gene known to cause Huntington’s disease. Their experiments offer not an immediate cure, ...
Jun 23, 2011
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New understanding of a gene that is linked to some forms of dementia and other age-related diseases gives scientists fresh hope that action can be taken against these diseases long before the onset of symptoms.
Jan 8, 2024
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Huntington's disease is caused by a mutation in the Huntingtin gene, a protein necessary for the proper functioning of several brain cells. Mutated, it is no longer able to perform properly: it can even become toxic for the ...
Mar 18, 2022
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More people may have the potential to develop Huntington's disease than previously thought, according to a study published in the June 22, 2016, online issue of Neurology, the medical journal of the American Academy of Neurology. ...
Jun 22, 2016
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Scientists have discovered how a gene mutation can lead to diseases that occur when the immune system attacks the body by mistake.
Nov 18, 2014
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Neurons often get most of the credit for keeping our brains sharp and functioning—as well as most of the blame when it comes to brain diseases. But star-shaped cells called astrocytes, another abundant cell in the human ...
Aug 30, 2022
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Heart disease kills more than 600,000 Americans every year, which translates to more than one in every four deaths. Although lifestyle choices contribute to the disease, genetics play a major role. This genetic facet has ...
Jun 7, 2017
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Sickle cell disease is an extremely debilitating condition that affects up to 40% of the population in African countries, with patients suffering episodes of excruciating pain, organ damage and reduced life expectancy. This ...
Jul 6, 2022
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In people, a baby's risk of congenital heart defects is associated with the age of the mother. Risk goes up with increasing age. Newborn mice predisposed to heart defects because of genetic mutations show the same age association.
Apr 1, 2015
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