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Presently, there is no effective treatment for the cancer found in the liver called fibrolamellar hepatocellular carcinoma, which is mainly found among children and young people. Operation of the tumour is the only treatment, some of the patients do not survive after five years.
Coupling of the two genes through a mutation causes a cancer tumour to develop in the liver. The researchers have made sure the genetic composition of the mutation in the mice is identical with the mutation found in human patients. This makes the researchers conclude that the gene mutation also leads to cancer in humans.
Sequencing the DNA of the patients tumour showed that all the patients suffering from the disease had the same mutation in their DNA. The researchers used the revolutionary technology CRISPR/Cas9 that makes it possible to ‘edit’ genetic material on mice.
They have produced CRISPR/Cas9 reagents, which would provoke precisely the desired fusion of the two genes.
The reagents were injected into the mice’s tails and then transported through the bloodstream to the liver.
In the liver they created a mutation identical with the human mutation, then the researchers were able to conclude that the mice developed the expected type of tumour in the liver.
In other studies, researchers deliberately damage the mice’s liver to imitate a liver disease found among human patients or expose them to several different forms of mutations which may cause cancer. This was not necessary here. Because of our genetic design based on CRISPR/Cas9-technology we are certain that the main factor that can explain the cancer is the fusion of the two genes.
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