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