November 18, 2016

CRISPR gene editing will be used in living humans to counter aggressive lung cancer

Chinese scientists have become the first in the world to use a revolutionary new gene-editing tool known as CRISPR-Cas9 on living humans.

Scientists at Sichuan University in Chengdu have injected a person with aggressive lung cancer with cells modified using the gene-splicing technology in a bid to make the patient’s immune system more effective at combating cancer cells.

It represents the first human trials of the technology with the United States expected to conduct its own cancer fighting trials next year.

CRISPR is a recently emerged technology that can be thought of as acting like a tiny pair molecular scissors that can cut and alter nucleotides which make up DNA, enabling scientists to find and modify or replace genetic defects.

The Chinese trial is expected to trigger a global race to carry out human trials of the groundbreaking medical technology throughout the world.

Gene-editing could improve the ability of immune cells to attack cancer.

Nature - CRISPR gene-editing tested in a person for the first time

On 28 October, a team led by oncologist Lu You at Sichuan University in Chengdu delivered the modified cells into a patient with aggressive lung cancer as part of a clinical trial at the West China Hospital, also in Chengdu.

Earlier clinical trials using cells edited with a different technique have excited clinicians. The introduction of CRISPR, which is simpler and more efficient than other techniques, will probably accelerate the race to get gene-edited cells into the clinic across the world, says Carl June, who specializes in immunotherapy at the University of Pennsylvania in Philadelphia and led one of the earlier studies.

CRISPR will target three genes in participants’ cells, with the goal of treating various cancers. The trial will start in early 2017. And in March 2017, a group at Peking University in Beijing hopes to start three clinical trials using CRISPR against bladder, prostate and renal-cell cancers. Those trials do not yet have approval or funding.

The researchers removed immune cells from the recipient’s blood and then disabled a gene in them using CRISPR–Cas9, which combines a DNA-cutting enzyme with a molecular guide that can be programmed to tell the enzyme precisely where to cut. The disabled gene codes for the protein PD-1, which normally puts the brakes on a cell’s immune response: cancers take advantage of that function to proliferate.

Lu’s team then cultured the edited cells, increasing their number, and injected them back into the patient, who has metastatic non-small-cell lung cancer. The hope is that, without PD-1, the edited cells will attack and defeat the cancer.



“The fear is that they could use these techniques to create, someway, genetically modified people. You know designer babies where parents pick and choose the traits of their babies, make them taller, stronger, smarter or something like that,” NPR reporter Rob Stein said in a recent report of a Swedish scientist using the technique to edit human embryos.

Despite the lack of understanding about the precise heritability of intelligence, it is plausible that CRISPR could be used to enhance the intellect of unborn babies, not just its physical traits.

“In my opinion, CRISPR could in principle be used to boost the expected intelligence of an embryo by a considerable amount,” James J. Lee, a researcher at University of Minnesota told Scientific American earlier this year.

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