Gene Therapy Gene correction:specific correction of mutant gene without other changes in target genome. Gene augmentation:modifying content or expression of defective cells by introducing foreign normal gene sequences
Gene Therapy ❖ Gene correction: specific correction of mutant gene without other changes in target genome. ❖ Gene augmentation: modifying content or expression of defective cells by introducing foreign normal gene sequences
Gene Therapy Goal b Somatic gene therapy Somatic therapy 与Transgene Transgenic dones
Gene Therapy ❖ Goal Somatic gene therapy
Gene Therapy Goal Somatic gene therapy b Germ cell gene therapy Injection of fertilized egg Transgene Egg
Gene Therapy ❖ Goal Somatic gene therapy Germ cell gene therapy
Gene Therapy Key b Safety concerns Effective expression
Gene Therapy ❖ Key Safety concerns Effective expression
Gene Therapy 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
❖ 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