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Liver-cell transplants show promise in reversing genetic disease affecting liver and lungs
Date:4/22/2011

April 21, 2011 -- (Bronx, NY) -- Transplanting cells from healthy adult livers may work in treating a genetic liver-lung disorder that affects millions of people worldwide, according to an animal study in the April 18 online edition of the Journal of Clinical Investigation. Jayanta Roy-Chowdhury, M.D. , professor of medicine and of genetics at Albert Einstein College of Medicine of Yeshiva University, is the study's senior author.

The genetic disorder, alpha-1 antitrypsin (AAT) deficiency, is the most common potentially lethal hereditary disease among Caucasians, affecting an estimated 100,000 people in the United States and 3.4 million people worldwide. AAT is a protein made by the liver that is essential for lung health. In AAT deficiency, the liver produces a misshapen form of AAT that cannot enter the bloodstream and instead gets stuck inside liver cells, causing two major problems:

  • AAT accumulates in the liver, leading to fibrosis (development of scar tissue) and liver failure;
  • Too little AAT reaches the lungs, where it's needed to rein in elastase, an enzyme produced by white blood cells. Elastase helps kill bacteria in the lungs, but uncontrolled elastase activity can damage lung tissue and lead to severe emphysema (chronic obstructive pulmonary disease).
  • In the study, Dr. Roy-Chowdhury and his colleagues tested cell therapy on transgenic mice whose liver cells (hepatocytes) had been engineered to produce mutant human AAT, resulting in liver fibrosis. When the mice were given infusions of hepatocytes harvested from the li
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    Contact: Kim Newman
    sciencenews@einstein.yu.edu
    718-430-3101
    Albert Einstein College of Medicine
    Source:Eurekalert

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