UC San Diego scientists have dramatically improved the success rate of genetically modifying human embryonic stem cells. This advance brings the promise of better treatment of genetic diseases.
The new approach works in 20 percent of embryonic stem cells, compared to less than 1 percent treated with standard methods, said Yang Xu, a UCSD professor of biology, who led the study, assisted by Hoseok Song and Sun-Ku Chung, postdoctoral fellows in his lab.
The study was published Thursday in the journal Cell Stem Cell.
Some genetic diseases can’t be studied adequately in animals, Xu said, so the ability to produce human cells with the diseases will be of great help. For example, drugs to treat the diseases can be tested in the genetically modified cells, he said.
Researchers transferred genes into the embryonic stem cells by using “bacterial artificial chromosomes,” circular pieces of DNA that resemble the circular DNA found in bacteria. The structure of these chromosomes make them more likely to introduce the genes into the cell nucleus than with standard methods.
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