322054 VO Human Gene Therapy: From basics to clinical applications - M14 (2024S)

Introduction to gene therapy, gene delivery vectors (viral, non-viral, physical delivery methods), expression vectors, molecular cloning, relevant immunology for the field, genome engineering, gene knockdown, genetic vaccinations, mRNA technologies, regulated transgene expression, vector-host interaction, principles of macromolecular delivery, examples of monogenetic diseases and gene therapy approaches, current pre-clinically and clinically evaluated and approved gene therapies and genetic vaccines, approved gene therapy approaches, cancer gene therapy, oncolytic vectors, CAR-T cell therapies.