MeSH tree: C16.320.400
Gene therapy has been successfully used to treat a number of diseases, including immune deficiencies, hereditary blindness, hemophilia and, recently, Huntington's disease, a fatal neurological disorder.
Feb 27, 2026
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MIT researchers have discovered that two common genetic mutations that cause Rett syndrome each set off a molecular chain of events that compromises the structural integrity of developing brain blood vessels, making them ...
Feb 25, 2026
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Neuroscience rarely enjoys clean experiments. Most brain disorders are mosaics of risk genes, aging, lifestyle and chance that leave their origins obscured. Huntington's disease (HD) is different. It begins with a single ...
Feb 18, 2026
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The brain's ability to do everything from forming memories to coordinating movement relies on its cells producing the right proteins at the right time. But directly measuring this protein production, known as translation, ...
Feb 18, 2026
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Numerous potential treatments for neurological conditions, including autism spectrum disorders, have worked well in lab mice but then disappointed in humans. What would help is a noninvasive, objective readout of treatment ...
Feb 11, 2026
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There is no known cure for Huntington's disease. A genetic mutation creates harmful proteins that accumulate and cause the disease's typical symptoms. A team from the Department of Human Genetics at Ruhr University Bochum, ...
Feb 8, 2026
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Olympic skiers, bobsledders and speed skaters all have to master one critical moment: when to start. As athletes prepare for the upcoming Winter Olympics, that split second is in the spotlight because when everyone is fast, ...
Feb 5, 2026
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A research team has successfully demonstrated the world's first gene-editing treatment for Leber's hereditary optic neuropathy (LHON). The study was conducted in collaboration with the Seoul National University College of ...
Feb 4, 2026
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The U.S. Food and Drug Administration has approved the Zycubo (copper histidinate) injection as the first treatment for Menkes disease in pediatric patients. Approval of Zycubo was granted to Sentynl Therapeutics.
Jan 21, 2026
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A team from the Faculty of Medicine and Health Sciences and the Institute of Neurosciences at the University of Barcelona (UBneuro) has applied advanced artificial intelligence techniques to better understand why Huntington's ...
Jan 20, 2026
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