322054 VO Human Gene Therapy: From basics to clinical applications - M14 (2021S)

Introduction to gene therapy, gene vectors (viral, non-viral, physical delivery methods), expression vectors, molecular cloning, relevant immunology for the field, genome engineering, gene knockdown, regulated transgene expression; vector-host interaction; principles of macromolecular delivery; examples of monogenetic diseases and gene therapy approaches; genetic vaccinations, 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.