Molecular Basis of Genetic Therapies

Catalog description

This course focuses on the basic science relevant to achieving efficient and effective gene transfer and genome editing in animal models and humans for the treatment of disease. The course includes units devoted to a variety of vectors useful for gene transfer, the fundamentals of genome editing, and current therapeutic approaches using specific diseases as models. Prior background in biochemistry, cell biology, and molecular biology is essential. Aspects of organ system anatomy and physiology, virology, and immunology that are relevant to the course material are included in the course. Because of rapid movement in this field, specific topics vary somewhat from year to year. Prerequisite: Required for all CAMB-GTV students. Non-CAMB students are not able to take this course, given the limited enrollment. CAMB students not in the GTV program must obtain instructor permission to take this course.