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
