A novel technique that lays down the groundwork for major advances in regenerative medicine, drug screening and biomedical research was created by researchers.
Using human pluripotent stem cells and DNA-cutting protein from meningitis bacteria, researchers from the Morgridge Institute for Research and Northwestern University were able to create a more efficient way to target and repair defective genes.
The team reported that the new method is much simpler than previous methods.
Zhonggang Hou of the Morgridge Institute's regenerative biology team and Yan Zhang of Northwestern University served as first authors on the study; James Thomson, director of regenerative biology at the Morgridge Institute, and Erik Sontheimer, professor of molecular biosciences at Northwestern University, served as principal investigators.
Hou said that with this system, there is the potential to repair any genetic defect, including those responsible for some forms of breast cancer, Parkinson's and other diseases.
He said that the fact that it can be applied to human pluripotent stem cells opens the door for meaningful therapeutic applications.
Zhang said the Northwestern University team focused on Neisseria meningitidis bacteria as it is a good source of the Cas9 protein needed for precisely cleaving damaged sections of DNA.
She said that they were able to guide this protein with different types of small RNA molecules, allowing them to carefully remove, replace or correct problem genes.
She asserted that this represents a step forward from other recent technologies built upon proteins such as zinc finger nucleases and TALENs.
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 said that 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.
He said that their collaboration with the Northwestern team has taken them further toward realizing the full potential of these cells because they can now manipulate their genomes in a precise, efficient manner.
The study has been published in Proceedings of the National Academy of Sciences.