322054 VO Human Gene Therapy: From basics to clinical applications - M14 (2018S)

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