Cure hereditary diseases with stem cells?

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Stem cells to heal an inherited metabolic disorder

British researchers have produced induced pluripotent stem cells (iPS cells) from the skin cells of patients with a metabolic disorder and then successfully converted them into healthy liver cells. The researchers then tested the healthy cells in animals on mice in order to cure the hereditary disease "alpha-1-antitrypsin deficiency".

This is a further step on the way to the medical use of iPS cells, even if the method has only been tested on mice so far, Allan Bradley and colleagues from the Sanger Institute in Hinxton, UK write in the current issue of the journal "Nature". Genetic changes that had led to cancer cells developing from the stem cells in earlier experiments had not occurred in the current trials, the British scientists continued. Since the iPS cells are ethically and morally less controversial than so-called embryonic stem cells, research places great hope in the medical use of artificially produced induced pluripotent stem cells.

Liver disease cured with induced pluripotent stem cells The scientists at the Sanger Institute, which is a non-profit genome research center in Hinxton nine miles south of Cambridge that focuses on developing the “genetic basis of health and disease”, have achieved significant success in the development of induced pluripotent stem cells. After researchers from the Hannover Medical School (MHH) and the Max Planck Institute for Molecular Biomedicine in Münster reported on the successful use of iPS cells to treat liver disease in mice in July, the British researchers have now been able to confirm these results. Allan Bradley and colleagues made iPS cells from skin cells from patients with a metabolic disorder, fixed the genetic errors responsible for the occurrence of the hereditary disease using two known methods, and then implanted the cells in mice suffering from the same metabolic disorder. In this way, Allan Bradley and his team reported that the hereditary disease "alpha-1-antitrypsin deficiency" was cured in the mice.

Alpha-1-antitrypsin deficiency "Alpha-1-antitrypsin deficiency" is an inherited disorder which, as a so-called monogenetic disease, leads to severe impairments of the metabolism. The disease is caused by the mutation of a single gene, the onset of which requires changes in both copies of the gene in question in the chromosomes of a patient. As Allan Bradley and colleagues report, about one in 2,000 people of Northern European descent develop "alpha-1-antitrypsin deficiency". The consequences of the hereditary metabolic disease can extend to cirrhosis of the liver, which in the worst case may require a liver transplant, according to the British researchers. Bradley and his team use a so-called jumping gene (transposon) and special enzymes, the zinc finger nucleases, to correct the genetic defects in the iPS cells obtained from the skin cells of three patients with the metabolic disease. The researchers then inserted the human cells into the liver tissue of mice with the same metabolic disorder. There the iPS cells took over the function of the defective liver cells and thus helped to free the mice from their inherited disease, the British scientists write in the journal "Nature". Apparently, the human cells integrated relatively smoothly into the liver tissue of the mice. In contrast to previous studies, no tumor cells were formed from the iPS cells during the current intervention, the researchers emphasized and added that the method could therefore be used in the future for the treatment of monogenetic diseases. However, further investigations are needed to analyze the mutations in the genetic makeup of the iPS cells that occurred during the study, even if no tumor cells have developed from them, Bradley and colleagues explained.

Criticism of stem cell research The induced pluripotent stem cells have been researched as a possible replacement for the highly controversial embryonic stem cells for years, with the reprogramming of stem cells from other cells being treated as an ethically sound alternative to the embryonic stem cells. The moral and ethical concerns have given embryonic stem cells an extremely bad reputation and have often been the subject of criticism. Because early human embryos have to be destroyed to produce the cells. Last but not least, the breeding of embryos in the laboratory (cloning) brought up by supporters of embryonic stem cells to generate the stem cells has triggered massive protests in public. In Germany, the dispute between opponents and supporters of embryonic stem cell research is also about when an embryo is to be protected as human life according to Art. 1 of the Basic Law. Comparable ethical discussions are of minor importance for iPS cells, but a controversy about possible health problems caused by the pluripotent stem cells (embryonic and induced) has flared up this year. U.S. scientists from the University of California, the San Diego School of Medicine and the Scripps Research Institute have identified serious genetic changes in pluripotent stem cell lines that, in the worst case, can lead to the formation of tumors. (fp)

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Image: Photo credit: Martin Gapa /

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Video: Stem Cells In Chronic Diseases. Roberta Shapiro. TEDxBeaconStreet

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