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Gene therapy shows promise in ARC syndrome, a deadly childhood liver disease

A new gene therapy has been used to successfully treat a deadly childhood liver disease in mice that model the disease, according to rese…

Source Medical Xpress AI Summary Updated 2h 28m ago

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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 cholestasis (ARC) syndrome is a lethal genetic disorder usually caused by a lack of the VPS33B protein, with children diagnosed with th…

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