Course Descriptions

Description: The goal of this course is to teach the molecular, developmental, and genetic bases of autism spectrum disorder and related Mendelian diseases (e.g., Fragile X, Rett, Tuberous Sclerosis, and Timothy syndromes). Each week, seminal papers will be discussed by Emanuel DiCicco-Bloom, MD, a developmental neurobiologist and child neurologist, and Jim Millonig, PhD, a molecular geneticist. Papers will be selected that have led to a greater understanding of the underlying pathology and the development of new treatments. A variety of cutting-edge techniques, such as immortalized pluripotent stem cells (iPSCs), will be introduced, and the positives and negatives of these approaches will be discussed. Each week, one disease will be examined, with Dr Millonig leading the molecular genetic aspects, while Dr DiCicco-Bloom will focus on the developmental studies. By the end of this mini course, students should have a greater understanding of these diseases, how scientific advances have led to the development of new treatments, and the challenges facing the generation of new therapies.