UBE3A gene replacement

**UBE3A gene replacement** is an investigational **AAV-mediated gene therapy** being developed by **Taysha Gene Therapies** for **Angelman syndrome**, a rare neurodevelopmental disorder caused by loss of functional maternal **UBE3A** in neurons. The therapy is designed for **cerebrospinal fluid or intrathecal administration** to deliver a functional **UBE3A** transgene to the central nervous system and restore neuronal UBE3A protein expression. Preclinical reports describe a construct that packages both the short and long UBE3A isoforms in an approximately endogenous **3:1 ratio**, with proof-of-concept activity in Angelman syndrome mouse models showing improvements in motor, behavioral, and seizure-related phenotypes. The program appears to have remained a **preclinical or research-stage** Taysha pipeline asset rather than an identified branded or clinically named product.

Other names

AAV-mediated UBE3A gene replacementUBE3A gene replacement therapyUBE-3A gene replacement therapyUBE 3A gene replacement therapy