- Research team have
identified a method of treating a cancer patient by silencing genes that code
for a protein called PD-1 using the CRISPR-Cas9 system.
trial was conducted to identify safety and has been successful thus far. The
trial will extend to 10 patients who will be reviewed after 6 months to
understand the effects.
- A similar trial was proposed by a
group of researchers from the U.S in June, creating a race between China and
the U.S for CRISPR-Cas9 system use in human trials.
cells, that used the CRISPR-Cas9 system, were injected into a patient with a
highly virulent type of lung cancer. This is the first time that this system
has been used on humans and it is also hoped that this method could soon be
used on patients with other types of cancer
who fail to respond to chemotherapy, radiation or other forms of
The CRISPR-Cas9 system was
one of the most sensational breakthroughs
of 2015 and this gene editing tool was meant to aid in a host of
disease conditions that involve genetic mutations
. Its use in mainstream
medicine may not be too far away, as reported by this recent study on the use
of CRISPR cas9 system on a cancer patient.
‘CRISPR-Cas9 system raises fresh hope of cancer cure after first human trial.’
This is a gene editing tool
that lets researchers
edit genes. It was first discovered by U.S scientist Jeniffer Doudna which led
her to win the breakthrough award for 2015.
tool has risen in importance and scientists from China and the U.S
have been trying hard to get it into mainstream medicine, where the tools can
be used to help develop a cure for genetic diseases.
Chinese Researchers Lead First
Ever Human Trial
Sichuan University's Lu
You isolated the immune cells from the blood of a lung cancer patient.
Immune cells express a protein called PD-1 that is a turn off switch to protect
the other cells in the body.
The CRISPR Cas 9 editing tool was used to
disable this protein so that immune cells can mediate an attack on the
The patient under study
is responding well to the treatment and a second injection may be administered.
Ten other patients may be selected for the study and followed up for a
period of 6 months and more to assess the safety of the trial.
By U.S Scientists
A similar research
project of about 250 million$ is being funded by Sean Parker's New Cancer
Institute and will be carried out at The University of Pennsylvania. This study
that was proposed in June was approved by the National Institute of Health but
approval from the Food and Drug Administration (FDA) is still pending.
This has raged a
biomedical battle between China and U.S in discovering mechanisms by which the CRISPR-Cas9 system can be safely used to treat cancer
and other life-threatening
conditions in humans.
Though this method of
treating cancers is expected to bring in laurels for the research group that
helps devise a meaningful and safe method, Chinese researchers have put their
foot forward first. The regulatory authorities in the U.S have more stringent
rules and the first step in the U.S trial is aimed at safety rather than
providing answers about its efficacy.
of the CRISPR-Cas9 System
of the System
- Unlike other editing methods,
CRISPR-Cas9 system relies on enzymes that are aimed at removing the target
- In the current model studied by the Chinese
researchers, genetically edited cells were injected into humans and the disease
brought under control. Though this study focused on treatment for cancers that
are difficult to treat, it may soon be used for all types of
- The system can be used to change the root cause of
the disease i.e the genetic mutation that resulted in the
- The gene editing could take place
in regions that are not the target regions, resulting in new mutations that
could lead to cancer.
- A previous study on human embryos
showed that out of 86 embryos tested
- Two-third had genetic
- 26 embryos only had the desired
Despite the drawbacks
associated with using this gene editing tool, it still holds a lot of promise
in treating various diseases.
System will be used by Editas Technology to treat gene-related blindness in humans.
- University of Stanford researchers plan to use the
CRISPR-Cas9 system to treat genetic mutations that lead to sickle cell anemia.
The CRISPR-Cas9 system
holds a lot of promise in treating genetic disease conditions in humans and the
first signs have already begun pouring in.
- First human treated with CRISPR gene-edited cells
in China, report says - (http://www.pbs.org/newshour/rundown/first-human-treated-crispr-gene-edited-cells/)