Researchers at the Broad Institute, The Jackson Laboratory, and the RARE Hope charity have successfully used the innovative prime editing technology and corrected five disease-causing mutations that cause alternating hemiplegia of childhood (AHC).

AHC is a rare and severe neurological disease that is accompanied by epileptic seizures and developmental delay.

These remarkable findings were published in the journal Cell.

As a result of this treatment, the symptoms of AHC were significantly reduced, and the life expectancy of treated patients was more than doubled compared to those who did not receive treatment. This is a major advance in the treatment of AHC, which is now a reality thanks to prime editing technology.

The method enables precise replacement of defective DNA segments without cutting the double helix, which reduces the risk of errors in the genome.

Also, researchers are exploring more non-invasive methods of treatment. And the possibility of using this approach at a later age enables expanding the possibilities of treating various rare genetic diseases.