Success in Development of Efficient Genetic Modification of ES Cells
Yang Xu, professor of biology at the University of California, San Diego and his colleagues Sun-Ku Chung and Hoseok Song have achieved success in development of efficient genetic modification of ES cells using the BACs. The breakthrough of the research group has brought the science of ES cells to a higher level. The technique they developed will enable rapid development of stem cell lines that can be used for both study of genetic diseases and testing of potential treatments.
The success in development of efficient genetic modification of ES cells technique bases on the use of BACs, synthesized circles of the human DNA which are replicated by the bacteria as their own chromosomes. The commercial BACs enable modification within the bacterial cells, while modified BACs have been shown to join with a correspondent section of human chromosome and replace sections of DNA when introduced to into human cells. The team has managed to replace altered genes in 20% of cells that were treated which is a major success considering that the standard techniques of genetic modification are successful in less than 1% of cells.
Xu’s team achieved success in transfer of a malfunctioned copy of p53, a gene that suppresses cancer into ES cell line. They were also successful with gene ATM. Mutated ATM is the cause of Ataxia telangiectasia, a severe inherited disease which leads to severe disability and increases the risk of cancer demonstrating the importance of efficient genetic modification of ES cells. Genetically modified mice with mutated copies of ATM do not share all traits of the gene with human patients and do not develop degenerated neurons. Thus the telomeres in mouse are long which makes study of accelerated shortening of telomeres in mouse impossible. It can be done only in the human cells.
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