This innovation presents a groundbreaking approach to addressing liver diseases.
Description
By leveraging human induced pluripotent stem cells (iPSCs), this technology enables the creation of engineered liver cells and tissues capable of gene-specific and genome-wide screening. This innovation holds significant potential for advancing drug discovery, disease modeling, and therapeutic interventions, particularly for conditions like nonalcoholic steatohepatitis and hepatocellular carcinoma.
Applications
• Nonalcoholic Steatohepatitis (NASH)
• Hepatocellular Carcinoma (HCC
• Drug Screening
• Gene Therapy
Advantages
Traditional animal models and immortalized cell lines often fail to accurately replicate human liver physiology and disease mechanisms. In contrast, the use of human iPSCs allows for the generation of liver cells and tissues that closely mimic human biology. The ability to perform gene-specific and genome-wide screenings in these engineered tissues provides a powerful tool for identifying key genetic drivers of liver diseases. Additionally, the technology’s versatility in creating both knockdown and knockout models enhances its utility in studying a wide range of genetic alterations. For example, the conditional knockdown of SIRT1 in iPS-derived liver cells has provided insights into fatty acid homeostasis and insulin resistance, demonstrating the platform’s potential for uncovering novel therapeutic targets.
Invention Readiness
The technology is currently at the in vitro development stage, with promising preliminary data supporting its efficacy. Engineered iPS-derived liver cells have been successfully created and used to model diseases such as nonalcoholic steatohepatitis and hepatocellular carcinoma. The next steps involve further validation of the technology through additional in vitro studies and potential in vivo applications. Collaborative efforts with industry partners and securing funding for advanced research will be crucial for advancing the technology towards clinical applications. The invention’s readiness for commercialization is bolstered by its strong scientific foundation and the growing interest in regenerative medicine and personalized therapies.
IP Status
https://patents.google.com/patent/US11866733B2
