Technology ID

Generation of Gene Specific Targeting Donor Plasmid (p5KI-GP-CHN and p3KI-GP-CHN)

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

This technology includes the use of engineered human induced pluripotent stem cells (iPSCs) for various applications such as studying cell differentiation, drug screening, and gene transfer therapy. It employs gene targeting donors flanked by DNA sequences compatible with endogenous loci to integrate transgenes through homologous recombination. A key aspect is the flexible gene targeting donor design, used in conjunction with safe harbor transcription activator-like effector nucleases (TALENs). These TALENs target specific endogenous loci, enabling high-efficiency and versatile genome engineering in human cells. Once the first-generation targeted cell lines are created using gene-specific TALEN, subsequent generations of isogenic cell lines can be produced using Cre recombinase. This method eliminates the need for repeated use of TALENs, thus enhancing re-targeting efficiency, reducing costs, and decreasing the genotoxicity associated with TALENs. The engineered reporter cell lines developed through this process are valuable for research in iPSC differentiation, transplantation, and drug screening.

Commercial Applications
The potential applications of this technology include advancing research in iPSC differentiation, transplantation, and drug screening, using engineered reporter cell lines.

Competitive Advantages
This technology offers competitive advantages by significantly enhancing re-targeting efficiency, reducing the costs and genotoxicity associated with the use of TALENs in genome engineering.
Licensing Contact:
Knezevic, Vladimir