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Docket #: S11-234

Robust and Sustained Transgene Expression with Mini-Intronic Plasmid Vectors

Researchers in Prof. Mark Kay's laboratory have developed a robust vector that combines the ease of plasmid preparation with the stable expression achieved by minicircle vectors. This technology – Mini-Intronic Plasmids (MIP) – integrates essential bacterial elements for antibiotic-free selection and propagation within an engineered intron contained within a non-coding exon. MIPs offer an easy to implement alternative to minicircles for a variety of gene transfer/therapy, protein production and research applications where optimal transgene expression is required. In many cases, transgene expression is up to 10-times higher than that achieved by routine plasmids, minicircles or when used within a viral vector such adeno associated viral vectors (AAV).

Stage of Research
The inventors have demonstrated up to 10-fold higher in vivo transgene expression from MIP vectors than minicircle vectors in mouse livers. Biological materials are available for evaluation.

Applications

  • Gene therapy - extrachromosomal expression of therapeutic genes without the risks associated with integration into patient genome
  • RNA and protein production - synthesis of peptides, proteins and RNAs
  • Robust AAV vector - robust transgene expression AAV vectors
  • Research - vectors for creating transgenic cells and animals

Advantages

  • Easy, scalable production:
    • standard plasmid preparation for mass production
    • RNA-out selection instead of antibiotic-selection
  • Robust, prolonged expression:
    • persistent expression at up to 10-fold higher levels than minicircles
    • no bacterial plasmid-induced DNA silencing
    • up to 10 fold enhanced AAV-mediated transgene expression

Publications

Transgene expression in mice


DNA constructs injected into mouse livers showed luciferase expression from MIP vectors was 7-fold higher than plasmid vectors and 3-fold higher than minicircle vectors.

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