Tonight, the Perpetual Notion Machine again explores the realm of gene editing. Five years ago this month, PNM talked with biomedical engineer Kris Saha about the process and techniques of gene editing. Borrowing an innate immunity mechanism in bacteria called CRISPR, and its Cas9 protein, scientists have been learning how to use, or essentially program, CRISPR to target specific genes to cut them from a DNA or RNA sequence. This ability has profound potential in treating inherited genetic diseases or disorders like Sickle cell anemia, Down’s syndrome, and Muscular dystrophy. And Kris has been working to enhance CRISPR for a specific disorder called Pompe. Pompe disease occurs in newborns and infants when glycogen cannot be broken down into smaller glucose sugar particles used by muscles for energy. This becomes life-threatening when the muscles are in the heart or lungs.
Also, Pompe disease has an added complication in that it occurs on multiple genes in different locations on EACH of the male and female chromosomes of the DNA sequences. But Kris and his team were able to program CRISPR to cut ALL the genes AT THE SAME TIME. Remarkable! But that’s not all . . . Since human testing was currently out of the question, and since no animal model could contain the exact human genome, computer modeling was used to test the CRISPR experiments.