Gene editing advance reverses disease in mice

Modesto Morganelli
Dicembre 8, 2017

"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.

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. "We were able to get around that concern". The Cas9 enzyme normally does the cutting, so the team used a dead form of it called dCas9 that can still target genes but doesn't damage them. These are coupled to the dCas9, along with the usual guide RNAs that help them locate the desired section of DNA. But the resulting protein-dCas9 attached to the activator switches-is too large and bulky to fit into the vehicle typically used to deliver these kinds of therapies to cells in living organisms, namely adeno-associated viruses (AAVs). To work around that issue, the researchers split the protein into two, loading dCas9 into one virus and the switches and guide RNAs into another. For muscular dystrophy, the researchers expressed genes that have been previously shown to reverse disease symptoms, including one particularly large gene that can not easily be delivered via traditional virus-mediated gene therapies.

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.

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.

"We were very excited when we saw the results in mice", said co-author Fumiyuki Hatanaka.

The Salk scientists adapted the regular CRISPR mechanism to influence gene activation without actually changing the DNA itself. The researchers plan to try to apply the technique to other cell types to help treat other diseases, and conduct more safety tests before human trials can begin.

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