Year 4 (NCE)
The overall goal of this project is to develop an induced pluripotent stem cell (iPSC)-based therapy for the treatment of stress urinary incontinence (SUI) in women. Our strategy consists of deriving iPSC lines with a non-integrative technology, differentiating lines to smooth muscle cells (SMC), assessing real-time characteristics of undifferentiated and differentiated cells, testing safety and efficacy in an animal model, and proceeding to initial discussions with the FDA about how to develop this product. During the previous reporting periods, we derived three patient iPSC lines, characterized the lines, optimized differentiation methods and established initial assays for analysis of undifferentiated cells, as well as established methods for animal studies. We have been successful in confirming SMC integration and survival in the animal model, as well as reproducible improvement in SUI in those animals. In addition we have established possible mechanisms of action of our target therapy and have filed a provisional patent application on our technology..