Genetics

CRISPR 'minigene' approach stops genetic liver disease in mice

A new CRISPR gene-editing technique prevented a genetic liver disease known to be driven by hundreds of different mutations and improved clinical symptoms in mice, Penn Medicine researchers reported in new proof-of-concept ...

Diseases, Conditions, Syndromes

Nonalcoholic steatohepatitis will pose clinical, economic burden

Nonalcoholic steatohepatitis (NASH) is expected to pose a significant clinical and economic burden during the next 20 years for U.S. patients with type 2 diabetes mellitus (T2DM), according to a study published online Jan. ...

Diseases, Conditions, Syndromes

Sox9 reshapes the biliary tree in Alagille syndrome

Alagille syndrome is a rare pediatric genetic disorder that can affect the liver, heart, kidneys, blood vessels, skeleton and other tissues. One major characteristic of this condition is abnormalities in the ducts that carry ...

Genetics

Genome editing helps decipher a congenital liver disease

Congenital hepatic fibrosis (CHF) is a rare genetic disease that causes malformation and fibrosis (scarring) of the liver. Occurring in roughly one out of every 20,000 births, CHF can lead to an enlarged liver, impaired blood ...

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