Scientists restore brain cells impaired by a rare genetic disorder
A therapy that restores brain cells impaired by a rare genetic disorder may offer a strategy for treating conditions like autism, epilepsy, and schizophrenia.
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.
The approach may help researchers develop treatments for other genetic conditions, including some that cause schizophrenia, epilepsy, ADHD, and autism spectrum disorder.
"It's immensely exciting because we now have the tools," says , a
