CRISPR gene-editing technique used in humans for the first time

For the first time, cells edited using the CRISPR-Cas9 technique have been injected into a human as part of a clinical trial involving a patient with aggressive lung cancer that was conducted at the West China Medical Center in Chengdu, according to a new report published by Nature.

A team led by Lu You at Sichuan University, also in Chengdu, delivered the modified cells into a patient on October 28, the journal revealed on Tuesday. They received approval from a hospital ethics review board in July, and were initially planning to inject the modified cells in August, but additional time was needed to culture and amplify the cells, delaying the procedure.

While clinical trials conducted using modified cells have been conducted before, The Verge said that this  marks the first time that scientists have used the easier “copy-and-paste” method known as CRISPR, which is easier to use, less expensive and can be used to grow and multiple cells far more quickly than other techniques.

The trial reportedly went well enough to warrant giving the patient a second injection of the cells and to expand the treatment to include nine additional patients, according to Engadget. After that happens, the patients will be monitored for at least six months to ensure that the injections do not cause any serious adverse side effects and that the treatment continues to work as expected.

Research demonstrates an ‘exciting strategy’ for treating lung cancer

Lu’s team removed immune cells from the patient’s blood and then used CRISPR-Cas9, which is a combination of a DNA-cutting enzyme and a programmable molecular guide, to disable the gene for the protein PD-1. PD-1, Nature explained, typically acts as a sort of checkpoint to turn off a cell’s immune response – a function that cancer uses to help itself spread.

The researchers then cultured these modified cells to increase their number, then injected them back into the patient, who has from metastatic non-small-cell lung cancer, the journal said. Thus far, Lu said that the procedure has been successful, and while the researchers were unable to give any detailed information due to privacy laws, the early results have scientists excited about the possibilities.

“I think this is going to trigger ‘Sputnik 2.0’, a biomedical duel on progress between China and the United States, which is important since competition usually improves the end product,” said Carl June, an immunotherapy specialist at the University of Pennsylvania who will serve as the scientific adviser for an upcoming US CRISPR trial expected to start in 2017.

Similarly, Naiyer Rizvi of the Columbia University Medical Center in New York told Nature that “the technology to be able to do this is incredible,” and Antonio Russo of Italy’s Palermo University added that using CRISPR to neutralize PD-1 in much the same way that antibodies have in the past is “an exciting strategy” with a “strong” rationale for treating lung cancer.


Image credit: Thinkstock

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