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

Article excerpt

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 the condition rarely living beyond their first year of life.