MeSH tree: C18.452.648.595
Researchers at the University of California San Diego and their colleagues have identified a key cellular pathway that drives brain degeneration in both a rare childhood disorder and the far more common Alzheimer's disease. ...
23 hours ago
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Lysosomes, the cell's recycling hubs, are best known for their role in a group of rare inherited conditions called lysosomal storage disorders, where harmful molecules build up inside these compartments. But scientists are ...
Jul 31, 2026
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How are disease-relevant molecules distributed within tissue? What changes occur even before they become visible under the microscope? Questions such as these are crucial for early diagnosis and targeted treatment in many ...
Jul 13, 2026
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Inside every human cell, proteins are constantly being tagged with small chemical modifications after they're produced. Known as post-translational modifications, or PTMs, these can change how a protein folds, where it travels ...
May 6, 2026
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An Australian-led international research collaboration has delivered a promising breakthrough in the quest to better understand and treat childhood dementia. Recently published in the journal Nature Communications, the study ...
Apr 7, 2026
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Ongoing clinical research at UNC could lead to a first-of-its-kind enzyme replacement therapy for Hunter syndrome, an ultra-rare disorder that causes progressive multisystem disease and neurologic decline.
Jan 5, 2026
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A team of researchers at the Broad Institute, led by gene-editing pioneer David Liu, has developed a new genome-editing strategy that could potentially lead to a one-time treatment for multiple unrelated genetic diseases.
Nov 19, 2025
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An evolving form of therapy to treat devastating neurodegenerative disorders by injecting fresh immune cells—microglia—directly into the brain, promises a new lease on health by slowing the progression of mind-robbing conditions.
Patients in a Phase I/II clinical trial conducted by UMass Chan Medical School of a dual vector gene therapy for GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases, exhibited a biochemical correction of the ...
Aug 15, 2025
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Tay-Sachs and Sandhoff diseases, genetic disorders affecting the brain, have no effective treatment and are typically fatal within the first years of life.
Aug 6, 2025
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