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 language | English (US) |
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Pages (from-to) | 843-857 |
Number of pages | 15 |
Journal | Molecular Therapy Methods and Clinical Development |
Volume | 17 |
DOIs | |
State | Published - 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+