MeSH tree: E02.095.301
University of Virginia School of Medicine researchers have used an advanced gene-editing technique to correct the underlying cause of a severe form of epilepsy in lab mice. This breakthrough could one day lead to new treatments ...
Apr 7, 2026
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Dr. Donald Kohn has been developing gene therapies for rare pediatric immune disorders for over 30 years. This week, his role in a clinical trial has culminated in the first-ever U.S. Food and Drug Administration–approved ...
Mar 28, 2026
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Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal muscle disease in children. The study shows in animal models that a single treatment is sufficient ...
Feb 26, 2026
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The Food and Drug Administration plans to drop its longtime standard of requiring two rigorous studies to win approval for new drugs, the latest change from Trump administration officials vowing to speed up the availability ...
Feb 19, 2026
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Viral mimic systems and other tech platforms could enable local testing of vaccine candidates and antiviral therapies. This is important in the context of low-resourced health settings, Africa's focus on increasing its vaccine ...
Feb 18, 2026
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Led by Mathieu Beraneck, researchers at the University of Paris Cité/CNRS and the University of Barcelona explored the strength of the relationship between a type of inner hair cell in the ear and balance. Their work is published ...
Feb 9, 2026
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A study by Brown University Health researchers has identified a crucial factor that may help improve treatment for glioblastoma, one of the most aggressive and common forms of adult brain cancer. The findings, published in ...
Feb 2, 2026
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Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the University of British Columbia are extending those possibilities to the skin for the first time. The UBC ...
Jan 27, 2026
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Serenity Cole enjoyed Christmas last month relaxing with her family near her St. Louis home, making crafts and visiting friends. It was a contrast to how Cole, 18, spent part of the 2024 holiday season. She was in the hospital—a ...
Jan 26, 2026
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A University of California San Diego-led team has discovered that restoring a key cardiac protein called connexin‑43 in a mouse model can dramatically improve heart function and extend survival in several inherited forms ...
Jan 26, 2026
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