Toggle light / dark theme

Scientists restore brain cells impaired by a rare genetic disorder

Posted in biotech/medical, genetics, neuroscience

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…


A therapy that restores brain cells impaired by a rare genetic disorder may offer a strategy for treating conditions like autism, epilepsy, and schizophrenia.