A study in The Journal of Cell Biology by scientists at the University of Massachusetts Medical School reveals important new details about the inner workings of the CRISPR-Cas9 machinery in live cells that may have implications for the development of therapeutics that use the powerful gene editing tool." We don't know a lot about the details of how the CRISPR-Cas9 complex gets around the genome of a live cell and finds its target," said Thoru Pederson, Ph.D., Vitold Arnett Professor of Cell Biology and Professor of Biochemistry and Molecular Pharmacology. "What we've learned in this study about how this machinery works is important and useful for gene editors looking to develop tools for the lab and potentially the clinic." A component of the bacterial immune system that protects it from viral invasion, the CRISPR-Cas9 complex is a powerful gene editing system. More efficient and precise than previous technologies, the CRIPR-Cas9 complex is being adapted in the lab, as scientists find ways to program and deliver it quickly to selectively edit specific genetic sequences for study. In order to cut a piece of double-stranded DNA, CRISPR-Cas9 makes use of a guide RNA made of roughly 20 nucleotides to target specific regions of a genome at which the Cas9 complex then makes the cut. This allows scientists to remove or insert genetic sequences into the genome. Because the underlying dynamics of how the CRISPR/Cas9 system works inside live cells aren't well understood, some delivery systems and techniques have been more successful than others. In order to observe the actions of the CRISPR-Cas9 system at work in a live cell, Dr. Pederson and colleagues developed a technique for labeling the guide RNA and Cas9 elements with different florescent molecules so they could be tracked simultaneously.
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