CRISPR: new technique for Gene-editing Therapy
1 December 2017 | Written by La redazione
The Anderson Lab team of the MIT finally created a nanoparticle system that envelops the CRISPR components in a protective sphere. A skin prick sends the treatment on its way to the liver—no blasting or virus required. In mice with genetically high cholesterol, the nanoparticles successfully turned off the faulty gene in over 80 percent of liver cells, bringing their blood lipid back to normal levels. That’s the highest success rate ever achieved with CRISPR in adult animals. “What’s really exciting here is that we’ve shown you can make a nanoparticle that can be used to permanently and specifically edit the DNA in the liver of an adult animal,” says Anderson.