The scientists then proved that the accurate copy of the gene was now active in the liver cells they had produced by demonstrating the presence of normal alpha1-antitrypsin protein in both test tube and mouse experiments.
"We have developed new systems to target genes and integrated all the components to correct, efficiently, defects in patient cells," says Professor Allan Bradley, Director Emeritus of the Wellcome Trust Sanger Institute. "Our systems leave behind no trace of the genetic manipulation, save for the gene correction.
"These are early steps but, if this technology can be taken into treatment, it will offer great possible benefits for patients."
"This study represents a first step toward personalised cell therapy for genetic disorders of the liver," explains Dr Ludovic Vallier, Medical Research Council (MRC) senior Fellow and Principal Investigator at the University of Cambridge's MRC Centre for Stem Cell Biology and Regenerative Medicine and Department of Surgery, who studies human pluripotent stem cell biology. "We still have major challenges to overcome before any clinical applications but we have now the tools necessary to advance toward this essential objective."
In their analysis of the stem cells, the research team showed that genomes of stem cells commonly contain mutations, whose cause remains unclear. However, the researchers were able to use the latest sequencing techniques to find cells with minimum numbers of mutations whose genetic consequences they could examine.
They make clear that careful screening of stem cells will be needed to contribute to safe a
|Contact: Don Powell|
Wellcome Trust Sanger Institute