24 March 2010: Lecture: Gene Therapy
After this lecture, you should be able to answer the following:
1. What is the basic rationale underlying gene therapy?
2. What is Leber's congenital amaurosis? How has gene therapy been used to treat the disease in dogs?
3. What are the different ways one can use to introduce DNA into human cells?
4. Describe an example of somatic cell genotype modification by DNA transfer.
5. Why are liposomes used for gene transfer? What are liposomes?
6. Describe the three categories of somatic cell gene therapy and provide at least one example of each.
7. List some of the advantages and disadvantages of using viral vectors to transduce DNA into patients. Are there differences among different viral vectors? How so?
8. Describe the success and the limitations encountered in the gene therapy of X-SCID disease. What kind of disease is this? What gene is affected? Do females get this disease? What seems to be the most significant adverse effect associated with the gene therapy of this disease?
9. Explain why gene therapy in the treatment of glaucoma is an area of intense investigation. Why would the introduction of genes into the eye which code for proteins such as MMPs, Prostagalandin Synthases, and Neuroprotectants make sense to use in the treatment of glaucoma?
1. What is the basic rationale underlying gene therapy?
2. What is Leber's congenital amaurosis? How has gene therapy been used to treat the disease in dogs?
3. What are the different ways one can use to introduce DNA into human cells?
4. Describe an example of somatic cell genotype modification by DNA transfer.
5. Why are liposomes used for gene transfer? What are liposomes?
6. Describe the three categories of somatic cell gene therapy and provide at least one example of each.
7. List some of the advantages and disadvantages of using viral vectors to transduce DNA into patients. Are there differences among different viral vectors? How so?
8. Describe the success and the limitations encountered in the gene therapy of X-SCID disease. What kind of disease is this? What gene is affected? Do females get this disease? What seems to be the most significant adverse effect associated with the gene therapy of this disease?
9. Explain why gene therapy in the treatment of glaucoma is an area of intense investigation. Why would the introduction of genes into the eye which code for proteins such as MMPs, Prostagalandin Synthases, and Neuroprotectants make sense to use in the treatment of glaucoma?
posted by Blase Billack, Ph.D. @ 11:27 PM
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