Developmental and epileptic encephalopathy (DEE) refers to a group of neurodevelopmental conditions characterized by developmental delay, cognitive impairment, and seizures in children. In 2016, the first case linking variants ...
Aug 29, 2023
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Researchers have developed an engineered adeno-associated virus (AAV) vector that yields high transduction of brain vascular pericytes and smooth muscle cells. The study describing the characterization of this novel AAV capsid ...
Aug 24, 2023
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Gene therapy that induces the body to create microRNA-22 (miR-22), a naturally occurring molecule, successfully treated mice with hepatocellular carcinoma, the most common form of liver cancer.
Aug 24, 2023
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A new study from the University of Minnesota is the first to demonstrate the ability for gene therapy to repair neural connections for those with the rare genetic brain disorder known as Hurler syndrome. The findings suggest ...
Aug 21, 2023
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A team of researchers has developed an inhalable vaccine that successfully protects against the COVID virus. It also opens the door to delivering other messenger RNA (mRNA) therapeutics for gene replacement therapy and other ...
Aug 20, 2023
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Crigler-Najjar syndrome is characterized by hyperbilirubinemia. If it is not treated quickly, the build-up of bilirubin, due to a deficiency in the enzyme UGT1A1 which is responsible for metabolizing it into a substance that ...
Aug 17, 2023
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Gene therapy might offer a one-time, sustained treatment for patients with serious alcohol addiction, also called alcohol use disorder, according to a new study led by a researcher at The Ohio State University Wexner Medical ...
Aug 14, 2023
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As medical technology advances, many diseases could be detected, prevented and cured with cells, rather than pills.
Aug 13, 2023
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A new study presents a promising treatment method for so-called fusion-driven cancers, which are currently often difficult to cure. These fusion-driven cancers are caused by an error in cell division that creates a fusion ...
Aug 11, 2023
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Researchers at the University of Bristol have made a remarkable step forward in finding a potential cure for a type of childhood kidney disease.
Aug 10, 2023
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