A gene-editing tool designed to precisely rewrite the gene that causes Huntington's disease reduced toxic protein fragments and symptoms associated with the disease in mice, researchers at the University of Illinois Urbana-Champaign ...
Jul 29, 2026
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Hereditary primary hemochromatosis is caused by a single faulty building block in a gene. This leads to iron overload, which can have serious consequences for organs and joints. In preclinical studies, researchers have already ...
Jul 8, 2026
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Research led by the University of Cambridge Loke Center for Trophoblast Research has shown that a genome-editing technique can be used to alter a single gene in human embryonic cells, enabling the study of very early human ...
Jun 25, 2026
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In 2025, baby KJ Muldoon became the first person to receive a personalized gene editing treatment, which likely saved his life. But the scientific advances that made the groundbreaking treatment possible were years in the ...
Apr 14, 2026
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Since 2017, a personalized immunotherapy called Chimeric Antigen Receptor, or CAR-T cell treatment, has worked wonders to treat patients with blood cancers such as leukemia. But when it comes to treating solid tumor cancers, ...
Apr 10, 2026
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Stem cell transplantation could be a rapid and effective way to restore hemoglobin production in individuals with the blood disorder β-thalassaemia. The treatment, presented in a phase 1 clinical trial, could reduce dependence ...
Apr 8, 2026
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University of Virginia School of Medicine researchers have used an advanced gene-editing technique to correct the underlying cause of a severe form of epilepsy in lab mice. This breakthrough could one day lead to new treatments ...
Apr 7, 2026
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Engineers at the University of Pennsylvania and Rice University have refined a technology for editing individual genetic "base pairs" to a new level of precision, opening the door to safer, more reliable therapies for a wide ...
Feb 23, 2026
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Mayo Clinic researchers have developed a promising gene-editing therapy that directly corrects a genetic mutation responsible for autosomal dominant polycystic kidney disease (ADPKD), the most common inherited kidney disorder.
Jan 29, 2026
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The human genome consists of around 3 billion base pairs and humans are all 99.6% identical in their genetic makeup. That small 0.4% accounts for any difference between one person and another. Specific combinations of mutations ...
May 21, 2024
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