I-Corps: NanoScript: A Nanoparticle-Based Artificial Transcription Factor for Effective Gene Regulation and Stem Cell Differentiation

The development of stem cells holds significant potential to transform the field of regenerative medicine by having the ability to treat and cure debilitating diseases. As such, there is an urgent need among biologists, researchers, and clinicians for a product that effectively regulates stem cell differentiation. To address this demand, the main goal of the proposed research is to develop a nanotechnology-based product (termed NanoScript). NanoScript is a tunable technology platform that effectively regulates gene expression in stem cells and differentiates stem cells into any cell lineage in a non-viral manner. NanoScript appeals to the broader stem cell market. Specifically, biologists and researchers, working at universities or private companies, who are interested in pursuing stem cell-based research are ideal customers, and are expected to be the 'early adopters.'With the advent of the proposed NanoScript technology, stem cells can safely and effectively be differentiated. By integrating small molecules onto gold nanoparticles, this team has developed NanoScript to mimic and emulate the function of natural proteins within the cell that regulate gene expression. The most attractive feature of NanoScript is its ability to regulate genes in a non-viral manner, which makes it a safe platform for fundamental and translational stem cell research. Furthermore, by making minor adjustments, NanoScript can be designed to induce the differentiation of stem cells into any cell type, thus making NanoScript a powerful and broadly-applicable platform. This interdisciplinary research and technology is the first-ever demonstration of a safe, effective, and tunable platform for stem cells.