High throughput derivation of disease-specific human induced pluripotent cell (hiPSC) lines: use of the [REDACTED] hiPSC selection and characterization platform to derive high quality, fully characterized pluripotent cell lines to support the CIRM iPSC de

Funding Type: 
hiPSC Derivation
Grant Number: 
ICOC Funds Committed: 
Public Abstract: 

Academic, pharmaceutical and biotech research has resulted in the development of incredibly effective medicines and therapies to treat disease. However, testing and developing potential therapies remains inefficient and costly. The process would be greatly improved with a method of accurately representing the diversity of human disease in a format applicable to drug identification. The recent discovery of Induced Pluripotent Stem Cells (iPSC) may allow the modeling of all human disease in a petri-dish. iPSC technology allows the “reprogramming” of any easily accessible cell type e.g. a skin cell or a blood cell, to become any other cell type in the body e.g. a brain cell, a heart cell or a pancreatic cell. Research has already shown the potential of this approach e.g. taking a skin cell from an individual suffering from a genetic disease such as Parkinson’s, reprogramming it to generate iPSCs, expanding these cells and then turning the cells into neurons (the dysfunctional cells responsible for Parkinson’s). In this way, a model of disease can be generated on which drugs can be tested. The current proposal is to use an industrial-scale iPSC technology previously developed in California to generate a stored bank of iPSC cells from thousands of individuals suffering from many different diseases. This bank will be a resource to researchers in universities and pharmaceutical companies who wish to develop models of human disease and identify more effective and safer drugs.

Statement of Benefit to California: 

The current proposal is to generate the world’s largest bank of Induced pluripotent Stem Cells (iPSC) from a list of specific genetic diseases. This activity would be directly funded by proceeds from the California Stem Cell Research and Cures Act – Proposition 71 and would fund California-based research and technology organizations. California has consistently led the world in high-tech and biotech innovation. The advent of iPSC technology has the potential to shape the future of drug development and regenerative medicine for the primary benefit of patients. The establishment of the CIRM iPSC bank will maintain California’s position as a center of excellence for biological research and specifically regenerative medicine. Moreover, work to establish and maintain the bank will not only actively employ skilled researchers here in California but will help support established and ongoing Californian research and industrial operations that have been active employers for many years. The effort will also require close collaboration between the Californian research, biotech and technology sectors, potentially leading to other, unanticipated technologies and therapeutic developments.