rAAV-EF1α​

The EF1α promoter (elongation factor 1 alpha promoter) is a highly conserved element derived from the gene encoding eukaryotic elongation factor 1-alpha, a key player in protein synthesis. Recombinant adeno-associated viruses (rAAVs) with EF1α promoters are extensively used in molecular biology and gene therapy due to their broad expression profile, stability, and robust performance in diverse systems.

Features of rAAV-EF1α

• Broad Expression:
  • The EF1α promoter drives high-level, constitutive transgene expression in a wide range of cell types, including neurons, glia, muscle cells, epithelial cells, and stem cells.
  • Effective in both dividing and non-dividing cells.
• Long-Term Expression:
  • Ensures stable, persistent transgene expression in vivo, ideal for chronic studies or long-term therapeutic interventions.
• Species Compatibility:
  • Functional in various mammalian species, including rodents, non-human primates, and humans.

Applications of rAAV-EF1α

• Gene Therapy:
  • Delivery of therapeutic genes to treat genetic disorders (e.g., muscular dystrophy, hemophilia, retinal degenerations).
  • Development of treatments for neurodegenerative diseases (e.g., Parkinson’s, Alzheimer’s).
• Neuroscience:
  • Expression of optogenetic tools (e.g., channelrhodopsin) for circuit mapping.
  • Delivery of fluorescent indicators like GCaMP or biosensors for real-time imaging.
• CRISPR/Cas9 Genome Editing:
  • Delivery of Cas9 and guide RNAs (gRNAs) for targeted gene editing.
  • Use of EF1α-driven systems for efficient expression in diverse tissues.
• Stem Cell Research:
  • Stable transgene expression in embryonic and induced pluripotent stem cells (iPSCs).
  • Differentiation studies and organoid development.

rAAV-EF1α is a versatile and reliable vector for gene delivery, offering robust, long-term expression across various cell types and species. It is widely employed in basic research, disease modeling, and gene therapy development. While not cell-type specific, its broad expression profile makes it a valuable tool for applications requiring general transgene expression.