Liver-Cell Transplants Show Promise In Reversing Genetic Disease Affecting Liver And Lungs

according to a recent animal study, transplanting cells from healthy adult livers may work in treating a genetic liver-lung disorder. The genetic disorder, alpha-1 antitrypsin (AAT) deficiency is the most common 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 major problems. In this 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 livers of healthy mice, the transplanted cells proliferated in the host liver, progressively replacing diseased hepatocytes. Most importantly, said Dr. Roy-Chowdhury, the transplanted cells reversed the fibrosis that had developed.