MeSH tree: C10.228.140.163
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 ...
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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As we age, the genes in our cells accumulate more mutations. This is one of the contributory factors to age-related diseases and the aging process. However, in a new study published in Science Advances, researchers have discovered ...
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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Ten years after the UK became the first country to legalize mitochondrial donation, the first results from the use of these high-profile reproductive technologies—designed to prevent passing on genetic disorders—have ...
Jul 19, 2025
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Scientists from the Centenary Institute and the University of Sydney have made a landmark discovery that could lead to safer and more effective gene therapies for a range of serious genetic disorders including Duchenne muscular ...
Jul 15, 2025
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An international research team has published a study in the journal The FEBS Journal that significantly advances the knowledge of a rare hereditary metabolic disease: classical homocystinuria. The team was coordinated by ...
Jul 8, 2025
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A research team led by Prof. Li Hai from the Hefei Institutes of Physical Science of the Chinese Academy of Sciences has developed a novel deep learning framework that significantly improves the accuracy and interpretability ...
Jul 7, 2025
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In May 2025, researchers announced that K.J. Muldoon, a baby boy born without the ability to process dietary protein properly, had become the first person to be treated with a customized gene-editing therapy. Based on a technology ...
Jun 25, 2025
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