Scientists from the Sanford-Burnham-Prebis Institute for Medical Research (SBP) have found that the effects of Alagille syndrome, an incurable genetic disease that affects the liver, can be reversed with a single drug. Research published Proceedings of the National Academy of Sciences.
More than 4,000 children are born each year with Alagille syndrome, which is caused by a mutation in the liver that prevents the formation and regeneration of bile ducts. Children with Alagille syndrome often require a liver transplant. Without transplant, the mortality rate from this disease in late adolescence is 75%.
Scientists have shown in animal studies that NoRA1 activates the Notch signaling pathway, which is impaired in Alagille syndrome. This triggered the regeneration and proliferation of duct cells in the liver, reversing liver damage and increasing survival.
The researchers are currently testing the drug on miniature livers cultured in the lab from cells from patients with Alagille syndrome. The new drug could also be used to treat certain types of cancer.
Source: Gazeta
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