MeSH tree: C16.320.565.398.641
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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Batten disease is a rare inherited condition that affects brain development and function. CLN3 disease is the most common type of this disease. The symptoms are life-changing. They usually begin between the ages of four and ...
Nov 6, 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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A study evaluating a pioneering lentivirus (LV)-mediated gene therapy trial for classical Fabry disease showed promising results over five years, indicating a potential breakthrough in treatment for the genetic disorder.
Jul 25, 2025
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When we hear the word "dementia," we usually think of memory loss in older adults. But there's another, much rarer form of the disease that strikes far earlier in life—childhood dementia, also known as Batten disease.
May 12, 2025
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If administered early, gene therapy has the potential to change the medical history of children born with metachromatic leukodystrophy (MLD), a rare and lethal neurodegenerative disease of genetic origin which leads to the ...
Apr 24, 2025
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In a preclinical study, researchers at Children's Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with potentially increased effectiveness and safety for the treatment of metachromatic leukodystrophy (MLD), ...
Mar 12, 2025
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In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and bone marrow transplantation—to alleviate symptoms of multiple sulfatase deficiency (MSD), an ultra-rare ...
Jan 29, 2025
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