Gene Therapy &o Understanding of the disease process 8 Structure/function of gene to be introduced g Efficient delivery of gene Control of gene expression g Prevention/control of immune responses Animal model and assessment of function Clinical trial❖ Understanding of the disease process ❖ Structure/function of gene to be introduced ❖ Efficient delivery of gene ❖ Control of gene expression ❖ Prevention/control of immune responses ❖ Animal model and assessment of function ❖ Clinical trial Gene Therapy