The CRISPR gene editing is used for the first time in American patients

The first clinical trials using CRISPR-published cells began in the United States. Researchers at the University of Pennsylvania are treating cancer patients with an experimental treatment, according to an NPR report.

CRISPR is a revolutionary tool for gene editing which can cut DNA with great precision, to disable genes, add new genes or change their functions. The gene editing technology is so powerful that it could be used to eliminate whole species or solve long-standing agricultural problems, such as annual slaughter of male chicks.

Two patients, one with multiple myeloma and the other with sarcoma, received treatment that uses a modified version of the patient's white blood cells. This is the first US example of using CRISPR to modify a specific type of immune cell "ex vivo" – outside the body.

The trial was launched in 2016 by the National Institutes of Health (NIH) and aims to recruit at least 18 participants to test the effectiveness of CRISPR treatment against three specific cancers.

The human immune system is a remarkable evolutionary adaptation that fights all kinds of invaders. In the fight against cancer, the results are not always so good. One of the reasons that cancer is particularly effective at escaping destruction and beating the "brakes" of the immune system.

In the test, the immune cells are removed from a patient and three modifications are made to the DNA by CRISPR. The modifications allow the cells to more easily attack a tumor, inhibiting the brakes and allowing the cells to fight cancer. Once the modifications are done in the laboratory, the patient's cells are reinjected into their body.

This is not the first time that CRISPR is used to edit human immune cells. A team of researchers from the Sichuan University in China is also working with CRISPR-modified immune cells to treat patients with non-small cell lung cancer. Preliminary results from this study suggest that the treatment is safe, but additional studies and larger samples will be needed to explore its effectiveness.

"This is only the beginning of the use of CRISPR for somatic editing," says Gaétan Burgio, a geneticist at the Australian National University, not associated with the trial. "We sometimes need to assess the safety and effectiveness of CRISPR over other available options."

CRISPR has been a wild month for humans. After the revelations of November 2018, the Chinese scientist He Jiankui CRISPR used to edit human baby embryos, the scientific community called for a moratorium on the edition of human embryos ("germ line edition") last March.

The critical difference between his work and the studies going on in the United States is when CRISPR does the editing. In the case of He, CRISPR has been introduced into the genes of an embryo, which will carry them throughout life. The clinical studies described here modify a set of cells that will eventually die, taking with them the CRISPR modifications. His work was very controversial and resulted in the termination of his employment, with a Chinese investigation calling his actions illegal and illegal.

Time magazine recently ranked He among the pioneers of his list of the 100 Most Influential People of 2019. His profile was written by CRISPR pioneer Jennifer Doudna, who said her work "broke scientific, medical and ethical "and" will probably remain in the memories as one of the most shocking applications of any scientific tool of our history ".