MeSH tree: C18.452.132
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 finally ...
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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In a historic medical breakthrough, a child diagnosed with a rare genetic disorder has been successfully treated with a customized CRISPR gene editing therapy by a team at Children's Hospital of Philadelphia (CHOP) and Penn ...
May 15, 2025
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Sugar—sweet, satisfying, and everywhere. From fresh fruit and honey to processed table sugar and drinks, it sneaks into nearly everything we eat. While delicious, sugar delivers what nutritionists call "empty calories"—energy ...
May 6, 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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