The ambitious idea of using CRISPR to cure genetic diseases before birth is one step closer to reality. Scientists reported on Monday that they used a form of the technology known as “base editing” to alter the DNA of laboratory monkeys in the womb, substantially reducing the levels of a toxic protein that causes a fatal liver disease before the animals had even been born.
The research, by a team at the University of Pennsylvania and the Children’s Hospital of Philadelphia (CHOP), will be presented next month at the annual meeting of the American Society of Gene and Cell Therapy, potentially paving the way for human trials.
But arguably the bigger deal, said study co-leader William Peranteau, is that CRISPR base-editing machinery, packaged in lipid nanoparticles, made it into a number of organs beyond the liver, including the heart, kidney, diaphragm, and skeletal muscles. “We were surprised to see that we were able to achieve moderate editing in some of these organs, which traditionally have been more difficult to access.”