BITC1311 Intro to Biotechnology
Chapter 11. Medical Biotechnology
Name
Thieman & Palladino Guided Notes
How are genes determined to be “homologues”?
What types of diseases involve apoptosis?
Describe why the Human Genome Project sequenced genomes from a wide range of organisms.
a. Did the HGP determine that humans have the largest number of genes of all organisms?
b. Under what conditions are genes called homologues?
c. What is apoptosis, and in what diseases is the study of apoptosis relevant?
d. How many genes do humans have in common with mice? With yeast?
4. Describe how a karyotype is prepared.
a. What is an advantage and a disadvantage of CVS over amniocentesis?
b. What type of diseases can a karyotype diagnose?
c. What is FISH, and how does it compare to a karyotype?
d. What type of diseases can a FISH analysis diagnose?
5. Describe how RFLPs can be used to diagnose a genetic disease.
a. What is a serious limitation to the general use of RFLPs in disease diagnosis?
b. How is ASO analysis done, and how can it overcome the limitations of the use of a RFLP for disease
diagnosis?
6. What is a SNP, and how frequently do they occur in the human genome?
a. Where in the human genome do most SNPs occur?
b. What is the mission of the SNP Consortium, and who are participating in it?
c. How do biotech companies hope to capitalize on the study of SNPs?
d. What technique provides the most rapid screen of SNPs?
7. How are protein microarrays made, and how can they be used to detect diseases?
8. Describe the roles played by oncogenes and tumor supressors in the formation of cancerous tissue.
a. How can knowledge about oncgenes and tumor suppressors be used in the war on cancer?
b. What is pharmacogenomics, and how does it allow for customizing medical treatments?
9. How are microspheres being used to improve drug delivery in the treatment of diseases?
10. What is nanotechnology, and how is it being applied to medicine?
11. What are some major advantages to being able to produce artificial blood? What limitations do current
approaches have in artificial blood products?
12. Describe some ways that MAbs might be used in the future to treat cancer and drug addictions.
13. What are the differences between ex vivo and in vivo gene therapy? Which of the two has been more effective
to date? Explain why.
14. Explain the difference between “transformation” and “transfection” of mammalian cells.
15. The problems with gene therapy can be dividing into three categories: delivery problems, expression
problems, and immune system problems.
a. What are some reasons that viruses make good vectors for gene therapy?
b. How are viruses engineered to make them safer and more reliable vectors?
c. What limits the use of “naked DNA” for transfection of cells and how can transfection efficiency be
improved?
d. What is a major drawback to the use of vectorless DNA for gene therapy?
16. What enzyme is missing in SCID patients, and what does this enzyme do in the body?
a. Describe the vector and strategy used to treat SCID with gene therapy. How effective has it been?
17. What are the symptoms of CF, and how does the molecular defect cause these symptoms to manifest?
a. How many different alleles have been discovered for this disease?
b. Describe the strategy that has been used to treat CF with gene therapy and discuss the success of this
approach.
18. Ho w many gene therapy trials have there been so far worldwide, and how many more are in the works?
a. How did Jesse Gelsinger’s death raise questions about the safety of gene therapy?
b. Discuss the expression problems that must also be addressed before gene therapy is successful.
1.
2.
3.
19. What is the new field of regenerative medicine about, and how does it differ from the type of medicine
practiced to date?
a. Parkinson’s disease affects 500,000 Americans and involves the loss of the substantia nigra of the brain.
How do drug treatments attempt to remedy this? About how long does this type of therapy succeed in helping
Parkinson’s patient? To what extent are patients improved from fetal neuron tissue transplants?
b. To what extent do tissue transplants offer recovery to people suffering paralysis from spinal cord damage?
c. What are the tissue typing proteins called, and how many genes are there in this family of proteins?
d. How is organ rejection avoided? What are the drawbacks to this strategy?
e. Why are pigs considered good candidates for xenotransplantation of organs? What makes GGTA1
knockouts useful for this?
f.
How are biocapsules used to overcome problems with tissue rejection?
g. Describe how “scaffolds” are made in tissue engineering.
20. Telomeres are tandem repeats of TTAGGG at the ends of chromosomes. What is their function?
a. Approximately how many cell divisions can most human body cells undergo before they senesce?
b. How do cancer and stem cells avoid this limit to cell divisions?
21. Describe the differences between cells called: gametes, zygote, embryo, morula, blastocyst.
a. Embryonic stem cells are most often harvested from the blastocyst stage of an embryo. Why are only the
inner cell mass of the blastocyst harvested?
b. What are the advantages of using embryonic stem cells, compared to other types of stem cells, for tissue
replacement?
c. How can parthogenesis be induced in eggs, and what advantage is there in using this technique in tissue
engineering compared to the use of embryonic stem cells?
d. What promise do adult-derived stem cells (ASCs) hold for replacing organ transplants?
e. Of what advantage is therapeutic cloning in tissue replacement, compared to ASCs? What makes
therapeutic cloning different from reproductive cloning?
f.
How successful have the attempts been to clone humans by nuclear transplant and by parthenogenesis?
22. One way to sequence a genome is the “shotgun” approach, where a random clonig process is used to produce
the fragments to be sequenced. Assuming that the maximum size of fragment that can be sequenced is 600
nucleotides long, what is the minimum number of fragments would have to be cloned in order to “cover” the entire
genome?
a. In the Human Genome Project (HGP), the different labs worked on an assigned chromosome of the human
genome in order to work in parallel with each other and to break up the genome into more manageable sizes.
What was the first step by which these labs break down each of their chromosomes into even more manageable
sizes?
b. How was a physical map of the human genome made at the beginning of the HGP?
c. What two approaches were used to make a genetic map of the human genome?
d. What technology is responsible for accelerating the completion of sequencing the human genome, giving
this project the distinction of being the only one of its size to come in ahead of schedule and under budget?