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.

Cells with excess UBE3A, an autism-linked protein, have atypical firing properties that can be corrected by limiting the protein’s levels, according to new research.

Two unpublished studies detail improved techniques for delivering gene therapies to the brain.

All five participants in a clinical trial of a gene therapy for Angelman syndrome experienced leg weakness, leading sponsors to pause the study.

Correcting an autism gene mutation in fetal mice lessens some autism-like behaviors after birth.