Updated on 20 August 2013
"We are able to guide this protein with different types of small RNA molecules, allowing us to carefully remove, replace or correct problem genes," Zhang said. "This represents a step forward from other recent technologies built upon proteins such as zinc finger nucleases and TALENs," Zhang added.
These previous gene correction methods required engineered proteins to help with the cutting. Hou said scientists can synthesize RNA for the new process in as little as one to three days - compared with the weeks or months needed to engineer suitable proteins.
Thomson, who also serves as the James Kress Professor of Embryonic Stem Cell Biology at the University of Wisconsin-Madison, a John D MacArthur professor at UW-Madison's School of Medicine and Public Health and a professor in the department of molecular, cellular and developmental biology at the University of California, Santa Barbara, says the discovery holds many practical applications.
"Human pluripotent stem cells can proliferate indefinitely and they give rise to virtually all human cell types, making them invaluable for regenerative medicine, drug screening and biomedical research," Thomson said. "Our collaboration with the Northwestern team has taken us further toward realizing the full potential of these cells because we can now manipulate their genomes in a precise, efficient manner," he added.
Sontheimer, who serves as the Soretta and Henry Shapiro Research Professor of Molecular Biology with Northwestern's department of molecular biosciences, Center for Genetic Medicine and the Robert H. Lurie Comprehensive Cancer Center of Northwestern University, said the team's results also offer hopeful signs about the safety of the technique.
"A major concern with previous methods involved inadvertent or off-target cleaving, raising issues about the potential impact in regenerative medicine applications," he said. "Beyond overcoming the safety obstacles, the system's ease of use will make what was once considered a difficult project into a routine laboratory technique, catalyzing future research," he added.