《医学遗传学 Medical Genetics》课程PPT教学课件（英文版）Therapy of the Hereditary Disease

Medica, Genetics Therapy of the Hereditary Disease

Therapy of the Hereditary Disease Medical Genetics

Operative Treatment Operation correction .&o Tissue and organ transplant

Operative Treatment ❖ Operation correction ❖ Tissue and organ transplant

Pharmacotherapy Rules

❖ Rules Pharmacotherapy

Dietetic reatment Rules

Dietetic Treatment ❖ Rules

Gene Therapy Gene therapy (is a technique for correcting defective gene responsible by transferring of a ftLrrctional normal copy of the gene into cel

Gene Therapy Gene therapy is a technique for correcting defective gene responsible by transferring of a functional normal copy of the gene into cells

Gene Therapy &o Gene correction: specific correction of mutant gene without other changes in target genome. g Gene augmentation: modifying content or expression of defective cells b y 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 Pu Somatic gene therapy Somatic therapy Transgene Transgenic don

Gene Therapy ❖ Goal  Somatic gene therapy

Gene Therapy Goal Pu Somatic gene therapy Pu Germ cell gene therapy Injection of fertilized egg Transgene Egg

Gene Therapy ❖ Goal  Somatic gene therapy  Germ cell gene therapy

Gene Therapy Key pu Safety concerns P Effective expression

Gene Therapy ❖ Key  Safety concerns  Effective expression

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