The approach, tested in mice, selectively boosts the expression of the autism-linked gene SCN1A in a subgroup of inhibitory cells.

Strategies to replace or compensate for mutated copies of the TCF4 gene could lead to treatments for this profound form of autism, a new study suggests.

A deactivated form of the gene editor restores UBE3A expression in mice and human neurons without cutting the genome. It may hold promise for future Angelman gene therapies.

The current system to approve genetic and metabolic conditions for newborn screening can’t keep pace with research on new therapies. Don Bailey wants to bring it up to speed.

A new viral variant can deliver genes exclusively to the brain, overcoming a key hurdle in treating neurological conditions using gene therapy.

Two new methods make it possible to delete long sections of the genome, expanding the reach of CRISPR gene editing.

A MECP2 gene therapy for Rett syndrome eases repetitive behaviors, anxiety and hyperactivity in a mouse model of Pitt-Hopkins syndrome.

After imposing a pause in November 2020, the U.S. Food and Drug Administration has cleared the way for a clinical trial of a gene therapy for Angelman syndrome.

A novel gene delivery system taps a protein found in people to encapsulate messenger RNA and transport it into cells.

Methods that selectively increase levels of the Rett protein make for safer and more effective treatment strategies, some researchers say.