Using a CRISpr/Cas10 genome editing technique known as a retrovirus targeting gene variant (sgRNA), scientists have developed a novel approach to treat a rare disease that affects the brain.
Their success has implications for many types of cancers, including gliomas, the most common form of brain cancer.
It has also raised concerns about how the virus may spread in the human population.
The procedure, called a gRNA-targeted retroviral delivery, uses the RNA (RNA-protein) to bind to a protein on the virus, which then copies the gRNA to the targeted gene.
The resulting virus then infects the cell.
For the brain, the procedure is relatively simple: it requires the targeted virus to be inserted into the brain tissue, which is then subjected to a process called retro-transplantation.
Although the technique has not been tested on humans yet, the team behind the research believes the potential benefits are significant, including potentially treating the brain cancer for which they have discovered a new treatment.
‘Treating the brain disease that causes Alzheimer’s disease with CRISP/Cas8 editing’A new study by the University of Sydney and Imperial College London has found that the CRISPA virus that causes brain tumors can be used to target genes in the brain of mice.
Using the CRispr/CISP-Cas9 gene editing technique, the scientists found that it is possible to edit genes in mice that are linked to brain tumors and then produce new tumors.
“This is the first report of CRISPr-Cas8-mediated editing of a tumor-associated gene in mice,” said Dr Daniel Hirschfeld, one of the paper’s authors and a research associate in the University’s School of Chemistry and Chemical Engineering.
Dr Hirschfield and his team, led by the Royal Victoria Hospital’s Dr Robert Mazzoni, have been researching the role of the gene in causing the disease.
They found that one of these gene variants, the TNF receptor gene, was expressed in the brains of mice that were infected with CRisps/Cas7, the virus that produces brain tumors.
“We found that TNF receptors are expressed in several brain regions in the mouse, including hippocampus and neocortex, and that these receptors may mediate the tumorigenic activity of the brain tumor,” Dr Hirschfeld said.
TNF receptor activity was found to be dependent on the genetic expression of the tumor-inducing protein TNF-alpha, which occurs in both brain tumors caused by the Cas7 virus and the normal brain.”TNF-beta has been found to mediate TNF activity in several other tissues and organs, including skeletal muscles and the pancreas,” Dr Mazzini said.
“This means that TnF-alpha may play a role in tumorigenesis.”
The researchers hope that this work could lead to novel approaches for the treatment of brain tumors, as well as other cancers, and help to identify targets for future therapies.
Scientists are already looking into ways to edit the genomes of other species, including humans, to target other gene variants that could be targets for cancer treatments.
With this research, they are using the same technique to target the Tnf receptor gene.
“It is a very promising discovery that is potentially very valuable to cancer researchers, as it could open up a whole new avenue of therapeutic targeting of the Tnnf receptor,” Dr K.N. Singh, who is a senior research associate at the Royal Melbourne Institute, said.
Dr Henschfeld, who has worked on the research for the past decade, said the study showed the utility of using CRISPS/Cas5 viruses to target gene variants.
He said the work is an example of the way that research is being done to explore the potential of CRisPS/Cis5CRISPS viruses to treat different types of cancer.
“What’s amazing about this is that you can target one gene variant and create a completely new tumor-suppressing effect in one species of animal,” Dr Singh said.