Invention Summary:
Since its introduction over two decades ago, the advancement of synthetic transcription factors has faced significant challenges, including poor membrane penetration, intracellular degradation, limited nuclear localization, low gene activation, and low synthesis efficiency.
Rutgers scientists have developed a novel synthetic transcription factor (STF) platform capable of efficiently regulating the central dogma of molecular biology in a safe, non-viral, and transient manner. These STFs are composed of biocompatible nanoparticle core with modular transcription factor domains to permeate the plasma membrane, targeting and entering the nucleus, and controlling transcription activity of endogenous target genes.
The NanoScript platform is an attractive alternative to conventional viral-mediated genetic manipulation, which may introduce random insertion of exogenous genetic material into the genome and increase the risk of mutation. The unique design of the STFs allows the separate synthesis of different components and functional domains, and tunable ratio among the DNA-binding domain, nuclear localization signal, and gene-modulation domain. The various components can be further optimized to enhance gene regulation control, modulate cytotoxicity, and prevent intracellular degradation.
Market Applications:
- Research tool for cellular gene expression modulation
- Gene and cell therapy reagent – cellular reprogramming for treating of spinal cord injury, hearing loss, diabetes, and more
Advantages:
- Efficient membrane penetration
- Precise gene targeting
- Excellent stability and biocompatibility
- Flexibility with additional targeting and moieties
- Ease of synthesis
Intellectual Property & Development Status:
US Patent 10,100,332, and allowed US application 16/437,898. Available for licensing and/or research collaboration.
