Technology ID

Generation of Safe-harbor Gene Targeting Donor Plasmids

Lead Inventor
Zou, Jizhong (NHLBI)
Rao, Mahendra (New York Stem Cell Foundation)
Research Materials
Development Stages
Pre-Clinical (in vitro)
Research Products
Human iPSC Lines
Lead IC

This technology includes two safe harbor gene targeting donors, specifically designed for applications in the study of induced pluripotent stem cells (iPSC). These include the pAAVS1D-CMV.RFP-EF1a.copGFPpuro and pAAVS1-iCLHN donors. A key feature of these donors is their ability to integrate various transgenes into specific loci through homologous recombination, facilitated by sequences homologous to safe harbor loci. When paired with TALENs targeting these loci, these plasmids enable precise and efficient genome engineering in human cells. Unlike existing AAVS1 gene targeting vectors, these donors offer the flexibility to target other loci and insert different transgenes, aided by built-in multiple cloning sites for easy cloning of homology sequences. Their potential applications are significant in iPSC research, specifically in areas like cell differentiation, transplantation, and drug screening

Commercial Applications
This technology's potential applications are notably impactful in the realm of iPSC research, offering advanced capabilities in differentiation studies, transplantation processes, and drug screening.

Competitive Advantages
The competitive advantages of this technology lie in its unique ability to target multiple safe harbor loci with high efficiency, coupled with built-in cloning sites for versatile transgene integration, setting it apart from existing AAVS1 gene targeting vectors.
Licensing Contact:
Knezevic, Vladimir