HomeCoursesMEDSCI 204: Pharmacology and ToxicologyLecture 26 Lecture 26 1. What is gene therapy? Delivery of nucleic acid that alters gene expressionWhat is gene therapy?== Delivery of nucleic acid that alters gene expression 2. What is somatic cell gene therapy? Therapy not passed onto offspringWhat is somatic cell gene therapy?== Therapy not passed onto offspring 3. What are advantages of gene therapy? "Long term effect, highly specific"What are advantages of gene therapy?== "Long term effect, highly specific" 4. What is ex vivo gene therapy? Genetic modification is performed in dish and transplantedWhat is ex vivo gene therapy?== Genetic modification is performed in dish and transplanted 5. What is in vivo gene therapy? Modification performed in patientWhat is in vivo gene therapy? ==Modification performed in patient 6. What are non-viral gene delivery vectors? "Plasmids, liposomes, siRNA"What are non-viral gene delivery vectors?== "Plasmids, liposomes, siRNA" 7. What are viral gene delivery vectors?== GM viruses 8. What does a GE virus carry? Gene expression cassetteWhat does a GE virus carry?== Gene expression cassette 9. What is the preferred virus for gene therapy?== Adeno associated virus (no disease associated with wild type virus) 10. What are gene therapy strategies? "Transgenes (enzymes, GFs, receptors), channel rhodopsins, designer receptors). RNA sequences (RNA interference, CRISPR gene editing"What are gene therapy strategies?== "Transgenes (enzymes, GFs, receptors), channel rhodopsins, designer receptors). RNA sequences (RNA interference, CRISPR gene editing" 11. What is gene therapy for PD? "Genes to synthesize dopamine in striatal cells, GDNF in striatum"What is gene therapy for PD?== "Genes to synthesize dopamine in striatal cells, GDNF in striatum" 12. What is cell therapy? Injecting cells into patientWhat is cell therapy?== Injecting cells into patient 13. What is autologous transplantation? Recipient is donorWhat is autologous transplantation?== Recipient is donor 14. What is synergenistic transplantation? Genetically identical donorWhat is synergenistic transplantation? ==Genetically identical donor 15. How do cell therapies work? Transplant cells release factorsHow do cell therapies work? ==Transplant cells release factors 16. What are antibody therapies? "Immunoglobulin specific for one antigen, usually IgH, humanised animal antibodies"What are antibody therapies?== "Immunoglobulin specific for one antigen, usually IgH, humanised animal antibodies" 17. What do antibodies do? "Bind ligands (interferes), receptors (blocks), antigens"What do antibodies do?== "Bind ligands (interferes), receptors (blocks), antigens" Loading...
