Technology ID

Generation of AAVS1 and C13 “Safe Harbor” Transcription Activator-life Effector Nucleases (TALENs) for Drug Screening or Gene Therapy Development

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

This technology includes AAVS1 and C13 “safe harbor” transcription activator-life effector nucleases (TALENs) for drug screening or gene therapy applications. TALENs are engineered sequence-specific DNA endonucleases that can significantly enhance genome-editing efficiency by >100-1000 folds. “Safe harbor” such as AAVS1 safe harbor and C13 safe harbor is genome locus that allows robust and persistent transgene expression with no or minimal interference of endogenous gene expression and cell properties. Construction of TALEN-expression vectors that specifically target safe harbor locus will create high-efficiency and widely-applicable system to achieve precise genome engineering in human cells.

Commercial Applications
Safe harbor, TALENs have broad applications on engineering any human primary cells and cell lines; safe harbor targeted human cell line, from either pluripotent stem cells or somatic cells, can be used for drug screening by reporter gene expression, or gene therapy by restoration of normal gene function.

Competitive Advantages
When used with safe harbor locus-specific gene targeting donor vector, AAVS1- or C13-TALEN will allow efficient insertion of cell-type specific reporter cassette or functional minigene into these safe harbors, and the transgene expression won’t diminish over the extended culture or differentiation.
Licensing Contact:
Knezevic, Vladimir