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

An estimated 30 million Americans are diagnosed with a rare disease. Before whole genome sequencing, it sometimes took more than a decade to find the specific genetic mutation.

Scientists report that gene therapy restored at least some hearing and speech for five out of six children with a rare form of genetic deafness.