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

Carola J. Maturana, Jessica L. Verpeut, Thomas J. Pisano, Zahra M. Dhanerawala, Andrew Esteves, Lynn W. Enquist, Esteban A. Engel

Research output: Contribution to journalArticle

1 Scopus citations

Abstract

Recombinant adeno-associated viruses (rAAVs) are used as gene therapy vectors. However, they have a limited payload capacity and lack small, long-lasting gene promoters for single-dose gene therapies. We identified small herpesvirus latency-associated promoters showing stable, pan-neuronal transgene transcription and translation in vivo for 6 months after AAV transduction.

Original languageEnglish (US)
Pages (from-to)843-857
Number of pages15
JournalMolecular Therapy - Methods and Clinical Development
Volume17
DOIs
StatePublished - Jun 12 2020

All Science Journal Classification (ASJC) codes

  • Molecular Medicine
  • Molecular Biology
  • Genetics

Keywords

  • AAV
  • Brain
  • CNS
  • Gene Delivery
  • Gene Promoter
  • Gene Therapy
  • LAP
  • LAT
  • Latency-Associated Promoter
  • Latency-Associated Transcript
  • Light-Sheet Microscopy
  • Recombinant AAV
  • Retro-Orbital Injection
  • Spinal Cord
  • Viral vector
  • iDisco+

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