Revolutionizing Transgenesis and Gene Disruptions using P-element Transposition
J.W. Larrick
Transomix, Inc., 2462 Wyandotte St., Mountain View, CA 94043, USA
Transposable elements have become an indispensable tool for research in
many organisms. The robust integration efficiency of the P element
transposon has significantly enhanced the capabilities of Drosophila
researchers. However, as the P element had been unable to integrate DNA
outside of the Drosophilidae family, this efficient transposon system
was unavailable for use in other organisms, until now. Modified P
element sequences have been engineered to enable this transposon system
to effectively integrate transgenes into a variety of cells in culture,
ranging from avian to human. The patented transposon system termed
StealthGene(C) can also deliver transgenes into whole animals including
mice, rats, goats and pigs. In mice and rats, StealthGene(C)
transposition leads to germline transmission. A high percentage of the
progeny contain and express the transposed transgene. A more recently
developed technology, Targeted Gene Delivery (TGD), utilizes the
modified P element in conjunction with specific genomic sequences from
the animal of choice and allows for site-specific gene integration for
transgenesis and gene knockouts. Together, StealthGene(C) and TGD
technologies enable the rapid alteration of genomes in mammals in very
powerful ways to accelerate the research of diseases such as
atherosclerosis, neurodegeneration , immune disorders and the processes
of aging. The potential for non-viral human gene therapy applications
to remedy such diseases in the future using these technologies may soon
become a reality.
Key words:
transposon, transgenic, knockout, gene, therapy
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