by Lauren Johnstone
CRISPR technology uses Cas proteins, most commonly Cas9,
found in bacteria bound to specific RNA molecules in gene editing. CRISPR is
already commonly used in scientific research and there is food available which
has been genetically modified using CRISPR. The hope is that this technology
could also be used in Medicine in treating and preventing disease. However,
there are some concerns about the safety and the ethics of the use of CRISPR.
Researchers have been investigating the safety of the
technology. The results from the first phase clinical trial of a CRISPR cancer
treatment were recently published in ‘Science’. The results of the trial were
promising regarding the safety of the technology. The trial involved removing
the immune cells of three patients with tumours with the same protein. CRISPR
was used to delete genes from these immune cells and a virus was used to add a
gene to the T-cells to ensure they targeted the specific protein found on all
three of the patients’ tumours. The cells were infused back into the patients.
The main concerns about the safety of using CRISPR in this
trial were the possibility that CRISPR would cause unintentional changes to the
genome that could result in cancerous cells and that the CRISPR protein used
for the gene editing may trigger an immune reaction. However, there were no
signs of the cells turning cancerous or any immune reaction and the patients
experienced no severe side effects.
There has also been research into genetically altering cells
so that they self-destruct in the presence of CRISPR. As many people have fears
about the uncontrolled spread of genetic mutations in the wild, this could potentially
be useful in helping prevent this. Engineering human cells so that they are
CRISPR-proof is also important to the safety of the technology if CRISPR is to
be more widely used in Medicine. At Harvard University a team engineered human
cells to produce guide RNA that would target LINE-1 DNA sequences. They also
gave the cells a gene for the Cas9 protein with a regulatory sequence that
meant it could only become active in the presence of doxycycline. When these
cells were added to a dish containing the antibiotic 99.9% of them died within
nine days. The hope is that this mechanism could be used in gene therapies,
much like the CRISPR cancer trial in the US, to shut the therapy down if any
problems are encountered. Though there are already other methods to manage the
safety of CRISPR the team from Harvard believe that theirs is better.
There is still more research and development taking place
using CRISPR which hopefully will reveal more about the technology and its
safety.
Sources:
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