The next step in editing genetic to treat incurable diseases

AFP

Thursday, 7 December 2017 20:17

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Thursday, 7 December 2017 20:17

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WASHINGTON | An advance in the edition genetics might allow treatment of incurable diseases such as diabetes or muscular dystrophies, show of recent animal experiments promising.

This new technique that boosts gene activity is a modified version of the method, Crispr/Cas9, a kind of molecular scissors.

While Crispr/Cas9 corrects genes by cutting accurately to the defective parts of the DNA, the new version allows you to selectively activate genes, say the researchers, whose work was published Thursday in Cell.

This approach eliminates the risk of change of genes by mistake, which is seen as a major problem, and potentially opens up the way for the use of the technique Crispr to treat human diseases devastating.

“Although many studies have demonstrated that Crispr/Cas9 can be used as a powerful genetic tool, there are increasing concerns about mutations in non-necessary generated by this technology,” says Juan Carlos Izpisua Belmonte, a professor at the Salk Institute, the lead author of the research.

“We have been able to overcome this problem,” he says.

To test this new technique, the researchers used mice in which they induced a disease severe renal, type 1 diabetes, and a form of muscular dystrophy.

In each of these cases, they have used the system Crispr/Cas9 in order to boost the expression of one or more specific genes that may eliminate the symptoms of these pathologies.

In the case of kidney pathology, scientists have enabled two genes known to play a role in the renal function, and have not only found increased levels of proteins related to these genes, but also an improvement in the functioning of these bodies.

For diabetes, they have activated genes that can generate insulin-producing cells, which resulted in a decrease of the blood glucose levels in diabetic mice.

As to the mice with muscular dystrophy, the researchers stimulated the activity of genes that have previously showed that they could reverse the symptoms of this disease. One of these genes, in particular, can not be easily routed by viruses, traditional way of genetic therapies.

“We have been very satisfied with the results obtained in these mice which showed that by inducing the activation of certain genes, we can at the same time to observe physiological changes,” says Fumiyuki Hatanaka, a scientist at the Salk Institute, one of the main co-authors of the study.