CRISPR Gene-Editing Injection Corrects Mutation in Liver Cells
The breakthrough method paves the way for correcting gene defects anywhere in the body. Most CRISPR-related treatments involve treatment of the cells in-vitro - outside the body and then injecting them into the target organ. Using CRISPR in-vivo - inside the body is a different ball game, requiring the transport of the whole editing assembly inside the cells. Researchers at Regeneron Pharmaceuticals and Intellia Therapeutics developed an injectable CRISPR treatment with a simple operation. They used messenger RNAs (mRNAs), which carry instructions for making proteins. Since the host cell can use the mRNAs to make its own proteins, researchers simply needed to send enough information to enable CRISPR editing inside the cell. To this effect, they injected two messenger RNAs (mRNAs), one to identify the mutation on the TTR gene and for making the Cas protein that can cut the DNA at the directed site. Since liver cells actively uptake foreign particles and are the site of origin for the d...