In August, chinese scientists will start the first human CRISPR trial

Hi dear followers,
 

I am back from my vacations and I see that I missed many exciting science news. And I missed you !

For my come back, I chose to feature the news from Nature: Chinese scientists are close to be the first worldwide to inject patients with cells modified using the CRISPR-Cas9 technique. 

Cancer cells in culture from human connective tissue. 

This image was released by the National Cancer Institute, 

an agency part of the National Institutes of Health, 

with the ID 2306

It is an exciting new step in the gene editing area…well in the cancer cure area. Indeed the scientists led by Lu You are planning to edit immune cells, T cells in order to increase their attack on tumors. The team will test in people who have metastatic non-small cell lung cancer.

The target gene is PD-1. The encoded protein PD-1 is a receptor expressed at the surface of activated T cells. It binds two ligands PD-L1 and PD-L2. Both are expressed by tumor cells. Blocking PD-1 using antibodies is described as a potential way to boost the immune response against tumor cells.

The goal of Lu's team is to knockout PD-1 using CRISPR-Cas9 and see an increased immune response against tumors.

I wonder if they are anticipating the off-target response: Not only that a PD-1 KO could affect immune cells metabolism in the whole body but also that the CRISPR-Cas9 by itself could lead to unintended KO genes. The team will extract T cells from the enrolled patients and then knocked them out. I wonder if a quality check will be performed and how ?

It seems like the team had the same questions!

They are going to try to extract T near the tumor site where they are already specialized. However, the lung-cancer tumors are not easily accessible so the team is hoping for a low rate of immune response.

To prevent to pick cells with gene editing in the wrong place, a company Chengdu MedGenCell will validate the cells to make that only PD-1 is KO.

As you can imagine, this CRISPR gene editing studies are a race. US is also planning to make a similar study, knocking out PD-1  and other gene target and knocking in a third one. US researchers think they could start at the end of the year.

A video from Newsy explain quite well the study.

If you want to read more:

• http://www.nature.com/news/chinese-scientists-to-pioneer-first-human-crispr-trial-1.20302