DBS 727: Foundation of Precision Medicine: from Genomics to Advanced Therapeutics

This graduate-level course provides a foundational overview of gene therapy and precision medicine, emphasizing the integration of genomic technologies with therapeutic design. Students
will examine nucleic acid chemistry, whole-genome sequencing, and variant interpretation, and introductory bioinformatics workflow through a dedicated laboratory component, as they relate to the diagnosis and treatment of rare genetic disorders and complex diseases. Core topics include gene augmentation, genome editing, RNA-based therapeutics, and viral and non-viral delivery systems, supported by case studies and critical analysis of preclinical and clinical data.
The course further includes a final, evaluated project in which students design and justify a therapeutic strategy for an inherited condition. Ethical considerations, regulatory frameworks, and
translational pathways governing advanced genetic therapies are also addressed. Upon completion, students will understand the scientific principles, technological platforms, and policy dimensions that shape modern genetic medicine.