151056550 GENE THERAPY ( 2 Crd.Hrs )

Pre-Requisites : 151056090

The course will discuss the modenrn approaches used in treating genetic diseases by introducing a normal functional gene in specific target cells or programing stem cells to copensate for the defective gene causing the clinical defect. The approach of using various vectors and other transfer stratigies to transfer the specific gene using specific models with their relative specificity, efficiency with their potential interference with the normal funcion of target cells will be thoroughly discussed with main focus on their potential mutagenesis and cell transformation effect. In addition, the various approaches used to suppress specific genes using interfering molecules or introduction of toxic genes to control cell transformation will also be discussed in details. The potential current and future stratigies used to repair defective genes by targeted recombination will also be introduced and discussed.