A Novel Gene-Editing Tool Could Treat Diseases Caused by Mutations

The tool could treat Huntington's disease and other conditions caused by mutations in genes.
Chris Young
A gene being treated by SATISalk Institute

SALK scientists have developed a gene-editing tool that could treat a wide range of diseases caused by mutations.

The tool, the researchers say, could bypass the need for the potentially damaging existing gene-replacement approaches by editing the gene while it is still in the process of being mutated.


Replacing existing approaches

Existing genome-editing approaches, unfortunately, often cause damage. The Salk Institute team says they have created a safer approach.

The new genome-editing technology,  recently described in a Cell Research paper, could be used to treat a wide range of gene mutation conditions such as Huntington’s disease and progeria.

The tool, dubbed SATI (Single homology Arm donor mediated intro-Targeting Integration), builds on HITI, a variant of the already established CRISPR-Cas9 gene-editing technique. 

As Engadget reports, CRISPR-Cas9 treats mutations by inserting a healthy copy of a problematic gene into the non-coding region of DNA. 

SATI, the team says, can perform edits on genes while the mutation is at work. While the DNA is repairing itself, the normal gene becomes integrated into the genome alongside the old one - this eliminates the troublesome gene without the risk of older procedures.

“This study has shown that SATI is a powerful tool for genome editing,” Juan Carlos Izpisua Belmonte, a professor in Salk’s Gene Expression Laboratory and senior author of the paper, said in a press release.

“It could prove instrumental in developing effective strategies for target-gene replacement of many different types of mutations, and opens the door for using genome-editing tools to possibly cure a broad range of genetic diseases.”

A work in progress

In lab tests, the new tool was shown to be very effective against progeria, a rare disease that causes premature aging.

Mice treated with SATI were shown to live about 45 percent longer while also showing reduced aging effects. The equivalent for a human affected by the same condition would be over a decade.

Of course, work is still needed, and the tool is still in the testing phase. The Salk team will now work on making SATI more efficient by trying to boost the number of cells that can incorporate the new DNA.

If SATI delivers on its promise, it could help doctors treat and eliminate a wide variety of deadly diseases caused by mutations.

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