Small Alphaherpesvirus Latency-Associated Promoters Drive Efficient and Long-Term Transgene Expression in the CNS

• Published in: Molecular therapy. Methods & clinical development Vol. 17; pp. 843 - 857
• Main Authors: Maturana, Carola J., Verpeut, Jessica L., Pisano, Thomas J., Dhanerawala, Zahra M., Esteves, Andrew, Enquist, Lynn W., Engel, Esteban A.
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 12.06.2020; American Society of Gene & Cell Therapy; Elsevier
• Subjects: AAV; Brain; CNS; Gene Delivery; Gene Promoter; Gene Therapy; iDisco; LAP; LAT; Latency-Associated Promoter; Latency-Associated Transcript; Light-Sheet Microscopy; Recombinant AAV; Retro-Orbital Injection; Spinal Cord; Viral vector; Brain; Viral vector; Gene Therapy; Gene Promoter; Latency-Associated Promoter; CNS; Recombinant AAV; Retro-Orbital Injection; iDisco; Latency-Associated Transcript; Spinal Cord; AAV; Gene Delivery; LAP; Light-Sheet Microscopy; LAT

Recombinant adeno-associated viruses (rAAVs) are used as gene therapy vectors to treat central nervous system (CNS) diseases. Despite their safety and broad tropism, important issues need to be corrected such as the limited payload capacity and the lack of small gene promoters providing long-term, p...