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.

A new spin on the classic CRISPR gene-editing tool can alter the genome’s chemical tags and suppress gene expression for months.

A new approach for delivering gene therapy to the brain reduces nerve damage in primates and could help make gene therapies for conditions related to autism safer.