Targeted Genome Mutagenesis using Active Purified PiggyBac TransposaseJHU REF: C10599
Invention novelty: A novel purified piggyBac transposase active in mammalian cells useful for an accurate, efficient transposon-mediated mutagenesis.
Value Proposition: Transposons have proven a powerful tool for genome engineering by insertional mutagenesis and transgenesis. However, their application is limited by the often-inaccurate cut and paste transposition, resulting in DNA duplications at the excision and target site, and the unavailability of active purified transposases, and there is great demand for new, more precise genetic tools. Hence, this technology provides a novel method for the fast and highly precise transposon-directed genetic manipulation of various different cells and organisms by administering an active purified piggyBac transposase. Advantages include:
- Injection of purified transposase avoids expression of the gene from RNA/Plasmid
- piggyBac catalyzes highly accurate transposition without footprint mutations at excision site
- Widely applicable for genetic manipulation and potentially gene therapy in various cells and organisms
Technical Details: Johns Hopkins researchers have isolated an active
Trichoplusia ni piggyBac transposase, valuable for the efficient and precise genome-wide mutagenesis in insect and mammalian cells. To date, the efficient use of transposons, mobile DNA segments that can change their position in the genome, as tools for genetic manipulation is impeded by the high frequencies of unwanted sequence duplications due to imprecise cut and paste transposition, and technical challenges arising from transposase expression from RNA or plasmid templates. Hence, this technology provides an active purified
Trichoplusia ni piggyBac transposase that can be directly injected into insect or different mammalian cells, thereby circumventing the need for expression from RNA or a plasmid. Intriguingly, piggyBac transposase, due to its unique target site usage, shows highly precise transposition and bypasses the necessity of DNA synthesis, thereby avoiding the creation of footprint mutations. Together, this presents an attractive new tool for the fast and efficient transposon-directed genome mutagenesis and possibly gene therapy in a variety of different cells and organisms.
Looking for Partners: To develop & commercialize the technology as a genetic tool for the rapid and precise genetic characterization and manipulation of insect and mammalian cells.
Stage of Development: Pre-Clinical
Data Availability: Under CDA / NDA
Patent Status: Tangible Material
Publication(s)/Associated Cases: EMBO J. 2008 Apr 9;27(7):1097-109.
