AAV-GT
UNC
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.
Pre-clinical
Discovery & Dev
Pre-clinical
Phase 1
Phase 2
Phase 3
To patients
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Disclaimer
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FAST Pillar
Therapeutic Approach
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.
Recent News
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Oct 2021
Learn MoreTaysha 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