CRISPR-Cas9 Gene Therapy Shows Promise in Preclinical Angelman Study

A CRISPR-Cas9-based gene therapy designed to increase the production of ubiquitin protein ligase E3A (UBE3A) — the enzyme lacking in Angelman syndrome — partially or completely corrected physical and behavioral deficits in a mouse model of the disease. The effects of a single administration of the therapy, when delivered before birth, were…

UBE3A Gene Reactivation Restores Neuron Physiology, But Not Behavioral Defects

Neuron activity is impaired in nerve cells located in a specific brain region of genetically engineered mice that lack the UBE3A gene. This activity can be restored upon UBE3A reactivation in the brain of adult animals, a mouse study reveals. The study, “Adult Ube3a gene reinstatement restores the electrophysiological deficits…