Within a large group of more than 700 patients treated with CAR T cell therapy, researchers found no evidence that the therapy itself caused any type of secondary cancer in the modified T cells, according to new analysis ...
Feb 3, 2025
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In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and bone marrow transplantation—to alleviate symptoms of multiple sulfatase deficiency (MSD), an ...
Jan 29, 2025
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Researchers at Washington University School of Medicine in St. Louis have described the neurodegeneration that occurs in the nervous system of the bowel in Batten disease, a rare and fatal genetic condition. In their latest ...
Jan 15, 2025
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In a finding with implications for one of the most promising cutting-edge medical treatments, researchers evaluated several measurement techniques commonly used in gene therapy. The study determined that one of the most popular ...
Jan 6, 2025
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Researchers at Children's Hospital of Philadelphia (CHOP) and the University of Pennsylvania Perelman School of Medicine pioneered a first of its kind gene therapy model that offers a potential breakthrough in treating X-linked ...
Jan 3, 2025
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Researchers from the Bakkers group at the Hubrecht Institute have successfully repaired damaged mouse hearts using a protein from zebrafish. They discovered that the protein Hmga1 plays a key role in heart regeneration in ...
Jan 2, 2025
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A novel drug holds promise for treating Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes severe muscle degeneration.
Dec 16, 2024
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New potential therapeutic targets have been identified for diabetic kidney disease (DKD)—the leading cause of kidney failure in the world—that could see patients treated with new gene and drug therapies preventing the ...
Dec 10, 2024
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A new gene therapy can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the amount of blood the heart can pump and dramatically improves survival, in what a paper ...
Dec 10, 2024
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Injured optic nerves can regrow toward the brain if treated properly, University of Connecticut scientists report in the September issue of Experimental Neurology. The findings in mice could lead to a potential treatment ...
Dec 4, 2024
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