UCLA scientists have devised a novel way to correct abnormal gene splicing – a common mutation that often leads to disease. A custom mask prevents the cell from seeing the genetic defect and restores the splicing to the correct location in the gene.
Using new technology made by Gene Tools of Oregon, the researchers designed a custom mask to camouflage each abnormal splice. The technique prevents the cell from seeing the genetic defects and restores splicing to the correct location in the gene.
The laboratory findings suggest a powerful new approach for treating cancer and other diseases caused by genetic mutations. The researchers? next step will be to test their findings in a mouse model.
Dr. Richard Gatti, professor of pathology and human genetics, and postdoctoral fellow Liutao Du at the David Geffen School of Medicine at UCLA, are available for interviews.
The research appears in the March 19 online edition of the Proceedings of the National Academy of Sciences.