MeSH tree: C10.228.140.163.100
Researchers have taken an important step toward solving one of the biggest challenges in gene therapy for children with metabolic liver disorders: growth itself. As a child's liver grows, some gene therapies that once worked ...
Sep 11, 2026
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How are disease-relevant molecules distributed within tissue? What changes occur even before they become visible under the microscope? Questions such as these are crucial for early diagnosis and targeted treatment in many ...
Jul 13, 2026
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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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Researchers at the Garvan Institute of Medical Research have uncovered a critical mechanism driving inflammation in mevalonate kinase deficiency (MKD), a rare but devastating autoinflammatory disorder. The study, published ...
Apr 30, 2026
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The need for medical treatments for rare diseases such as Leigh syndrome is high. However, low patient numbers make research into treatments difficult. Together with a team from the University of Luxembourg, researchers from ...
Apr 20, 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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Chinese researchers have developed a novel and highly efficient mitochondrial capsule transplantation therapy, achieving the safe and efficient transplantation of healthy mitochondria into cells and tissues for the first ...
Mar 23, 2026
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Sildenafil—an active ingredient also marketed under the name of Viagra—improves symptoms in patients with Leigh syndrome. This has now been reported in the journal Cell by researchers at Charité—Universitätsmedizin Berlin, ...
Mar 11, 2026
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Virginia Tech researchers have discovered an indication hidden in the brain that may help doctors identify children suffering from a rare genetic disease earlier. Their findings are published in EMBO Molecular Medicine.
Jan 28, 2026
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The U.S. Food and Drug Administration has approved the Zycubo (copper histidinate) injection as the first treatment for Menkes disease in pediatric patients. Approval of Zycubo was granted to Sentynl Therapeutics.
Jan 21, 2026
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