MeSH tree: E05.393.420
At the beginning of July 2026, the U.S. Food and Drug Administration approved the first gene therapy for young children with sickle cell disease.
Jul 15, 2026
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A gene therapy designed to replace the missing protein that causes fragile X syndrome restored several disease-relevant traits in a mouse model, according to a new study published in Gene Therapy.
Jul 15, 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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A new Europe-wide survey launched during the 42nd Annual Meeting of the European Society of Human Reproduction and Embryology (ESHRE) suggests broad public support for fertility treatment and several areas of reproductive ...
Jul 4, 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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"Peacemaker" immune cells could help treat diseases ranging from type 1 diabetes to neurodegeneration by restoring immune tolerance, according to a new paper in Frontiers in Science.
Jun 25, 2026
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The availability of genetic testing for amyotrophic lateral sclerosis (ALS) for people with a family member diagnosed with the disease is expected to greatly increase the number of clinic visits to specialized ALS centers ...
Jun 24, 2026
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A gene therapy designed to replace a missing brain protein restored normal brain activity and improved behavior in a mouse model of fragile X syndrome (FXS), according to a study led by researchers at the University of California, ...
Jun 20, 2026
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A study from the University of Calgary shows that removing a single gene makes colon cancer cells a target for immunotherapy—a fundamental breakthrough. The research, published in Cell Reports Medicine, was led by Dr. Arshad ...
Jun 19, 2026
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A new gene therapy has been used to successfully treat a deadly childhood liver disease in mice that model the disease, according to researchers at UCL and Great Ormond Street Hospital. Arthrogryposis, renal dysfunction and ...
Jun 19, 2026
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