Taysha Gene Therapies Announces Publication of Positive Proof-of-Concept Preclinical Data for an AAV-mediated UBE3A Gene Replacement Approach Demonstrating Therapeutic Potential for The Treatment of Angelman Syndrome in the Journal JCI Insight

Angelman syndrome (AS) is a monogenic neurodevelopmental disorder caused by deletions or mutations in the maternal ubiquitin protein ligase E3A (UBE3A) gene.