The ASPIRE Path in Molecular Medicine

My proposed thesis research focuses on Steroidogenic Factor-1 (SF-1), a nuclear receptor crucial for steroidogenic tissue development and function. As an orphan nuclear receptor, SF-1’s precise mechanistic understanding is incomplete, yet its dysfunction is implicated in conditions like adrenocortical carcinoma and endometriosis. Recent mass spectrometry data associates sphingolipids with SF-1 in carcinoma cells, though structural confirmation remains elusive. My project will elucidate the structural and functional consequences of sphingolipid binding to SF-1, a critical step toward understanding its full regulatory capacity. While this research is fundamental, its direct implications for developing targeted small molecule therapeutics are significant. The progression from basic mechanistic insight to clinical application involves preclinical animal models and ultimately human clinical trials-areas vital to drug development. My current doctoral work provides a strong foundation in early-stage drug discovery. The APMM program’s emphasis on clinical research education offers the ideal environment for my project. The potential for my SF-1 research to transition from fundamental discovery to therapeutic application will serve as a practical framework, enabling APMM students and post-doctoral fellows to envision and plan the translation of their own basic research into human subjects studies.