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PiggyBac transposon system for Gene Therapy

Effective gene therapy techniques require in vivo gene transfer and expression of a target gene. PiggyBac (PB) has been used to deliver gene therapy targets to the genome in vivo. Since only small increases in expression are required to alleviate the bleeding phenotype; human factor VIII (hFVIII) deficiency is an enticing target for gene therapy. In animal models for hemophilia, PB introduced hFVIII in vivo and drove stable expression of the transgene for more than 6 months, thereby correcting the hemophilia phenotype.

Partnering Strategy
Transposagen Biopharmaceuticals, Inc. holds worldwide exclusive rights for the use of piggyBac (PB) for almost all commercial applications, including human therapeutics. Exclusive and nonexclusive licenses are available through Transposagen. If you have an interest in sub-licensing piggyBac technology, please email ordering@transposagenbio.com or call 859-428-8561.

�Embryonic stem (ES) and iPS cell engineering and differentiation

�Immunotherapy

�Stable Expression of transgenes in cultured cells