University of North Carolina, in collaboration with Taysha Gene Therapies, is exploring an investigational Adeno-Associated Virus - Gene Therapy (AAV-GT) dual isoform approach to treating Angelman syndrome.
A gene modulation technology that utilizes a nuclease-deactivated Cas9 protein that binds to the target genomic region with the same efficiency as Cas9, but does not cut the DNA and instead can exert RNA-directed transcriptional control of the targeted gene with a goal to upregulate a specific gene.
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
