MicroRNA Regulation in Stem Cells, Development, and Disease

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MicroRNA Regulation in Stem Cells, Development, and Disease

Public Abstract:
Pluripotent embryonic stem cells (ESCs) have the remarkable capacity to differentiate into any specialized cell type in the human body and are therefore of potential therapeutic value for neurodegenerative diseases, spinal cord injury, diabetes, heart disease, and numerous other degenerative diseases. Moreover, several of these same stem cell pathways are hyperactivated in tumorinitiating/ cancer stem cells. Therefore identifying novel gene regulatory pathways required for both the growth of these unique cells, as well as their differentiation into specialized cell types is key to developing novel therapeutic approaches for the treatment of degenerative disease and cancer. Our goal is to further develop a specialized, high-impact research program focused on understanding the mechanisms of gene regulation in pluripotent stem cells. With this information, we will design and develop assays to identify new drugs that regulate the differentiation of stem cells into specialized cell types and that suppress tumor growth by eradicating tumor-initiating/cancer stem cells. Our comprehensive and integrated research program will maximize the likelihood of realizing our goal of identifying chemical compounds that can specifically control cellular fates for therapeutic use.
Statement of Benefit to California:
To fully benefit from the potential of regenerative medicine to treat injury and degenerative diseases we need to be able to effectively control and manipulate cellular fates on demand. This will require the successful translation of basic knowledge gained from understanding the molecular mechanisms controlling stem cell differentiation into specific cell fates, such as specialized cells of organs (e.g. insulin producing pancreatic beta cells) or specific types of neurons. In order to more effectively accomplish this, it will be necessary to understand the molecular circuitry that operates in stem cells and bestows these remarkable cells with their unique ability to become any cell type. My laboratory has been successful in identifying novel stem cell pathways that regulate this fundamental capacity of stem cells and that can be targeted therapeutically. In the short term, the benefit to California will be the relocation of my specialized research program to the state, and, with the support of CIRM, the further development of this world-class, high-impact research program, which will perfectly complement the existing research strengths in genomics, RNA biology, and stem cells at the University. In the longer term, the citizens of California will benefit from my laboratory’s efforts aimed at the identification of new drugs that will target these crucial, stem cell pathways and stimulate the body's own repair mechanisms. We anticipate that these new drugs will heal diseased tissues and organs previously considered irreparable. Moreover, our research has the potential of identifying new chemotherapeutic agents that target cancer stem cells, a recently identified cell that is likely to be responsible for incidences of tumor recurrence following treatment. The State of California will benefit by being at the heart of these clinical advances and the resulting economic impact as they translate to the market.