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.
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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
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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
