Immune-system cells play important roles in a wide range of diseases from diabetes and AIDS to cancer. Using a popular genome-editing system, scientists from University of California-San Francisco have for the first time devised a new strategy to precisely modify human T-cells, also called immune cells. This discovery opens new avenues for therapies based on the genome-editing system known as CRISPR/Cas9 for many serious health problems. The gene-editing system has captured the imagination of both scientists and the general public as it makes it possible to easily and inexpensively edit genetic information in virtually any organism.
Alexander Marson, senior and co-corresponding author of the study, said, "Genome editing in human T-cells has been a notable challenge for the field. There are a lot of potential therapeutic applications."
Using their novel approach, the researchers were able to disable a protein on the T-cell surface called CXCR4, which can be exploited by HIV when the virus infects T cells and causes AIDS. The research group also successfully shut down PD-1, a protein that has attracted intense interest in the burgeoning field of cancer immunotherapy. Using drugs to block PD-1 coaxes T cells to attack tumors.
T-cells not only stand at the center of many disease processes but can also be easily gathered from patients, edited with CRISPR/Cas9, then returned to the body to exert therapeutic effects. An enzyme, called Cas9, in the CRISPR system that makes cuts in DNA and allows new genetic sequences to be inserted, has generally been introduced into cells using viruses or circular bits of DNA called plasmids.
The research was published in the Proceedings of the National Academy of Sciences.