Discussion
Scientists in the U.K. have created a liver-specific gene therapy that could potentially treat arthrogryposis renal dysfunction cholestasis (ARC) syndrome, a fatal childhood disease marked…
Cholestasis
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Cholestasis is a medical term that’s used to describe a slowing or stalling of bile flow out of the liver. Bile is a yellow-greenish fluid produced by the liver that helps to break down certain fatty molecules and proteins into smaller pieces during digestion. It also carries certain waste products from the liver into the intestines to be excreted in feces.
Cholestasis treatment options aim to restore the normal flow of the digestive fluid bile through the bile ducts, a series of tubes that carry the sticky yellow-greenish fluid out of the liver where it's made and to the small intestine.
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A liver transplant with an uncle serving as a living donor successfully managed advanced progressive familial intrahepatic cholestasis type 2 (PFIC2) in a one-year-old…
Discussion
Children with progressive familial intrahepatic cholestasis (PFIC) who carry mutations that completely eliminate a protein’s production are nearly three times more likely to require…
