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.
In this edition of Null and Noteworthy, tests of intranasal oxytocin and an Angelman syndrome treatment fail to see results.
As treatments for some autism-linked genetic conditions inch closer to the clinic, researchers are talking more urgently about screening all newborns for such conditions.
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.
A gene-editing treatment shows long-lasting effects in a mouse model of Angelman syndrome, a genetic condition related to autism.
All five participants in a clinical trial of a gene therapy for Angelman syndrome experienced leg weakness, leading sponsors to pause the study.