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Gene Editing: CRISPR Human Trials in US Due to Start

Dec 15, 2016 11:10 AM EST
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A little girl named Layla was treated with gene-edited immune cells in 2015. This helped eliminate all signs of leukemia that were out to kill her body. It was experimental, but by the end of 2017, many lives would have been saved with this revolutionary gene editing treatment.

As explained in New Atlas, gene editing involves altering or disabling genes already existing in the body. This proved to be extremely difficult before, but only if looking at it from the wrong angle. 

It took years to develop, but the method used to save Layla, called the CRISPR, is as revolutionary as it is effective. It could be done in weeks and with astonishing results.

According to Engadget, the first human trial involving the method began in China. It is being used to disable a gene called the PD-1 in immune cells taken from individuals with cancer. The edited cells are injected back into the patients' body. PD-1 has an "off switch" on the surface of immune cells. This is useful for cancers as they eventually get an ability to flip the PD-1 to switch "off," thus rendering the immune system useless.

On the edited cells, there is no switch to turn off, thus making the immune system more efficient.

According to New Scientist, the trial in the US is already underway, and it appears to be more ambitious as ever. The new trials will add an extra gene that was engineered to make the immune cells target tumors as well. CRISPR will then be used to disable the PD-1 and two other genes.

Previous tests involving the addition of tumor-targeting genes have already shown promise for trials of cancers such as leukemia. However, it has not worked well for solid tumors yet. It is hoped that combining the two will make treatments more effective. 

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