MeSH tree: C18.452.132.100.937
Scientists at Pacific Northwest Research Institute (PNRI) have overturned a long-held belief in genetics: that inheriting two harmful variants of the same gene always worsens disease. Instead, the team found that in many ...
Jan 12, 2026
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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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The liver is the body's control tower for metabolism, powering vital functions like converting nutrients to glucose, storing fat and breaking down toxins. Over a third of the world, however, is thought to be affected by conditions ...
Apr 16, 2025
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By exploiting the technology used in COVID-19 vaccines, a team led by UCL, King's College London and Moderna scientists has created an effective therapy for a rare disease, in a study in mice, demonstrating the technology's ...
Jan 10, 2024
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Ammonium is a biomarker used to diagnose a series of rare hereditary metabolic disorders, such as the primary disorders of the urea cycle and different organic acidemias, as well as other metabolic and environmental conditions ...
Nov 3, 2022
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