MeSH tree: E05.393.420.301
Some of the symptoms of neurodevelopmental disorders (NDDs), such as autism spectrum disorder and epilepsy, may result from dysfunction of certain neurons in the brain and their ability to change their properties in response ...
Aug 12, 2026
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A 6-year-old Chinese girl died last year after receiving an experimental gene therapy to correct a non-life-threatening condition, according to an investigation by Science and Retraction Watch.
Jul 24, 2026
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Sickle cell disease causes premature aging of blood stem cells, which scientists may be able to address with a special class of drugs, according to a new study from St. Jude Children's Research Hospital. Patients with sickle ...
Jul 23, 2026
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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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"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 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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Muscle spasticity is a common and often debilitating consequence of spinal cord injury that can significantly affect mobility and quality of life. Spasticity is a condition in which muscles become abnormally stiff or tight ...
Jun 9, 2026
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