Studying diseases with better delivery of gene-editing tools penn_state
A Penn State–led team of interdisciplinary researchers developed a method that improved the lifespan and efficiency of CRISPR gene-editing tools after delivery into stem cells. Their findings were made available online ahead of official publication in"We want to study diseases that arise from an inherited trait, but examining the diseased cells can be challenging," Lian said.
To design their new delivery system, Lian's team leveraged an enzyme that would allow for long-term integration of the gene-editing tools. With a conventional method that relies on DNA components to deliver the tools into cells, they can survive and edit genes for about three days before being destroyed, Lian said.
After a seven-day waiting period, the researchers assessed the results. They found that the PiggyBac system had successfully—and permanently—integrated the tools into the cells' genome, and that 99% of the cells tested had mutated to block the expression of the targeted protein. This PiggyBac delivery method led to a much higher number of mutated cells than traditional DNA delivery methods, which resulted in around 10% of cells blocking the protein, Lian said.
"If we couple together stem cell technology and gene-editing tools as we have in this research, we can quickly study diseases without asking a patient to donate their