Scientists restore brain cells impaired by a rare genetic disorder

https://www.npr.org/sections/health-shots/2024/04/30/ [login to see] /timothy-syndrome-genetic-disorder-brain-cells-restored
Scientists have found a way to restore brain cells impaired by a rare and life-threatening genetic disorder called Timothy syndrome.

A type of drug known as an antisense oligonucleotide allowed clusters of human neurons to develop normally even though they carried the mutation responsible for Timothy syndrome, a team reports in the journal Nature.