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Stem cell advance offers prospect of cure for genetic liver diseases

By Sharon Kingman
Staff Writer

Friday, December 6, 2013
LONDON – Physicians will soon attempt to treat patients with liver diseases caused by single-gene defects, by performing highly specific gene therapy on single liver stem cells, then growing up the genetically engineered cells and transplanting them back into the liver via an injection in the portal vein. The method could be applied to many different single-gene disorders, so long as a technique exists to grow the stem cells from the affected tissue that needs treating. Although the study that provides proof that the method works was carried out using stem cells from patients with cystic fibrosis, researchers are not currently planning clinical studies focusing on cystic fibrosis.

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