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Use Of CRISPR In First Human Trial Given Green Light In The US

DNA helix
If it goes ahead, it will be the first human CRISPR trial. ktsdesign/Shutterstock

The gene-editing technique that has taken the scientific world by storm, CRISPR, will undergo its first clinical trial. This week saw an advisory committee for the US National Institutes of Health unanimously approve a trial that will use CRISPR to edit a type of human immune cell in order to engineer it to better fight cancer.

The first trial to take place will not be looking explicitly at whether or not it can help to tackle the cancer, but instead will be looking at whether or not the technique is safe for use in humans. The trial, which will be undertaken by scientists at the University of Pennsylvania, will be funded by the immunotherapy foundation created by cofounder of Napster and first president of Facebook, Sean Parker.


This first trial will only be small, involving a total of 18 patients, who each have one of three types of cancer. The researchers will remove some of their T-cells, a part of the immune system that is responsible for hunting down and destroying cancer cells, and perform three edits on them using CRISPR. The first will add a gene that codes for a protein that has been engineered to detect cancer cells. The second edit will remove a particular T-cell protein that might interfere with the newly added gene, and a third will remove another protein that cancer cells use to identify the cells as T-cells. These edited cells will then be infused back into the patients.

As well as looking at how safe CRISPR is for use in people, the trial will also assess how feasible it is to manufacture genetically engineered cells. The idea of altering human cells has long worried some people, but opinion has slowly been changing as many questions of safety and ethics have been answered. This year, for example, saw the UK as the first country to allow for the genetic engineering of a human embryo, but under the caveat that they will be kept for no longer than 14 days.

CRISPR-Cas9 has revolutionized genetics over the past few years: The method enables the rapid and yet precise targeting of DNA, which can then be cut and other genes added in. It is thought that with the ease and speed at which this can be achieved, it will completely revolutionize the treatment of genetic diseases. 


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