Huntington's disease is a hereditary neurodegenerative disorder caused by a gene on chromosome 4 that affects a very important area of the brain, the striatum. People are born with the defective gene but symptoms do not appear ...
Feb 20, 2020
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Mice born blind have shown significant improvement in vision after undergoing a new gene therapy developed by a team of Japanese scientists.
Feb 7, 2020
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The causes of rare diseases could be uncovered using an approach created to identify genetic mutations that trigger a muscle-wasting condition, a study suggests.
Dec 18, 2019
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Scientists at the Institute of Pharmacy at Martin Luther University Halle-Wittenberg (MLU) have developed new delivery vehicles for future gene therapies. A team of researchers led by Dr. Christian Wölk are using artificial ...
Nov 18, 2019
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In experiments with rats, pigs and monkeys, Johns Hopkins Medicine researchers have developed a way to deliver sight-saving gene therapy to the retina. If proved safe and effective in humans, the technique could provide a ...
Sep 30, 2019
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U.S. regulators have approved the most expensive medicine ever, for a rare disorder that destroys a baby's muscle control and kills nearly all of those with the most common type of the disease within a couple of years.
May 24, 2019
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Scientists at the Children's Medical Center Research Institute at UT Southwestern (CRI) are using a new approach to pinpoint the causes of rare genetic diseases in children and identify treatment options faster than with ...
Apr 30, 2019
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A new study from BC Children's Hospital, the University of British Columbia (UBC) and an international team of researchers published in the New England Journal of Medicine is the first to identify a rarely-seen type of DNA ...
Apr 15, 2019
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Many diseases are caused by genetic defects. However, their severity can vary among individual patients, so that even mild forms can occur. Scientists at the Max Planck Institute for Heart and Lung Research in Bad Nauheim ...
Apr 10, 2019
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In addition to humans, hereditary disorders of enamel development occur in dogs, greatly impacting their dental health and wellbeing. A recent study reveals canine enamel disorders similar to those found in humans, linking ...
Apr 1, 2019
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