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iPSC model aims to increase understanding of liver disease

Researchers from Boston University and Boston Medical Center have developed a patient-derived induced pluripotent stem cell model that could be used to better understand liver and lung disease, according to study data published in Stem Cell Reports.

“We hope that the insights we gain from these studies will result in the discovery of new potential treatments for affected patients in the near future,” Andrew A. Wilson, MD, assistant professor of medicine at Boston University School of Medicine and director of the Alpha-1 Center at Boston University and Boston Medical Center, said in a press release.

Andrew A. Wilson

Wilson and colleagues from Children’s Hospital of Philadelphia and other institutions, created induced pluripotent stem cells (iPSC) from normal patients (controls) and patients carrying mutations (PiZZ) in the gene responsible for alpha-1 antitrypsin (AAT) deficiency and exposed them to growth factors in-vitro. This process led them to turn into liver-like cells, according to the research.

Analyses showed that the iPSC cells secreted AAT protein slower than normal cells and were also more sensitive to certain drugs compared with normal cells.

“This is important because it suggests that the livers of actual patients with this disease might be more sensitive in the same way,” Wilson said in a press release. “This approach might now be used to generate that sort of evidence to guide clinical decisions.”

According to the research, the PiZZ transcriptome diverges from controls upon reaching hepatic stage, when AAT is first expressed, providing potential clues to liver pathogenesis. PiZZ iPSC-hepatic cells model key liver disease features and demonstrate both therapeutic drug responsiveness and sensitivity to toxic agents. – by Melinda Stevens

Disclosure: The researchers report no relevant financial disclosures.