Lifelong elimination of hyperbilirubinemia in the Gunn rat with a single injection of helper-dependent adenoviral vector

What is it about?

Researchers demonstrated that is possible to cure the animal model of a inherited liver disease known as Crigler-Najjar syndrome. In this genetic disease the lack of a liver enzyme causes the accumulation of unconjugated bilirubin which, untreated, results in severe jaundice and brain damage. Current therapy relies on phototherapy to reduce unconjugated bilirubin levels. Due to the beneficial effect of light exposure, the symbol of Crigler-Najjar patients is the sunflower. Gene therapy might represent a potential alternative to current therapeutic approach. This study shows that a single administration of a gene therapy vector containing the human gene which is defective in the disease can lead to life-long correction of the bilirubin levels in the animal model of the disorder.

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Photo by Khoa Ma on Unsplash

Photo by Khoa Ma on Unsplash