《医学遗传学》课程PPT教学课件（留学生 Medical Genetics）11 Chapter Therapy of the Hereditary Disease

MedicalGenetics Therapy of the Hereditary Disease

Therapy of the Hereditary Disease Medical Genetics

Operative Treatment Operation correction Tissue and organ transplant 101

Operative Treatment ❖ Operation correction ❖ Tissue and organ transplant

Pharmacotherapy Rules 003

❖ Rules Pharmacotherapy

Dietetic Treatment *Rules

Dietetic Treatment ❖ Rules

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

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