FINDINGS: UCLA scientists have devised a novel way to correct abnormal gene splicing – a common mutation that often leads to disease. Splicing edits out the punctuation from a cell’s genetic blueprint, enabling its code to dictate protein production by the cell. 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.
IMPACT: 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.
AUTHORS: 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.
JOURNAL: The research appears in the March 19 online edition of the Proceedings of the National Academy of Sciences.
Proceedings of the National Academy of Sciences