Generation of Safe-harbor Gene Targeting Donor Plasmids

Description:

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

Patent Information:
For Information, Contact:
Vladimir Knezevic
Senior Advisor for Commercial Evaluations
NIH Technology Transfer
301-435-5560
vlado.knezevic@nih.gov
Inventors:
Jizhong Zou
Mahendra Rao
Keywords:
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