Human Heredity: Principles and Issues (MindTap Course List)
11th Edition
ISBN: 9781305251052
Author: Michael Cummings
Publisher: Cengage Learning
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Chapter 16, Problem 13QP
The prospect of using gene therapy to alleviate genetic conditions is still a vision of the future. Gene therapy for adenosine deaminase deficiency has proved to be quite promising, but many obstacles remain to be overcome. Currently, the correction of human genetic defects is done using retroviruses as vectors. For this purpose, viral genes are removed from the retroviral genome, creating a vector capable of transferring human structural genes into sites on human chromosomes within target-tissue cells. Do you see any potential problems with inserting pieces of a retroviral genome into humans? If so, are there ways to combat or prevent these problems?
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4) List 3 things that a potential vector must have in order to be useful.
5) Sometimes the gene for the enzyme glutathione-S-transferase (GST) is used in purifying your
protein of interest. Briefly explain how this is done.
6) What enzyme is used to create CDNA from MRNA?
In the treatment of acquired immunodeficiency syndrome (AIDS), a possible mode of therapy is to inhibit the reverse transcriptase (RT) of the human immunodeficiency virus (HIV), whcih is required for the retrovirus to be propogated by RNA-directed DNA synthesis.
In the figure below, one of the substrates for RT is thymidine; and two drugs, AZT and HBY097 are known to inhibit HIV RT>
(a) Thymidine; (b) AZT; (c) HBY097
Look at the structures and predict the type of inhibition (i.e. competitive or non-competitive) likely to be shown by each drug.
By using knowledge on enzyme, plan an experiment that would enable you to confirm the type of inhibition by investigating enzyme kinetics and explain how you would interpret the results.Remarks: Not more than 250 words.
Retroviruses may transform cells in more than one way. From the figure given below,
explain the two ways by which cells are transformed.
You have the purified protein overexpressed in the cell. How will you determine which pathway
of expression is occurring in the cell?
Promoter
V-onc
Retroviral DNA -
Retroviral DNA
Retroviral Proto-
promoter oncogene
Proto-
V-onc
oncogene
Host cell DNA -
Host cell DNA
Excessive
Lexpression
Expression
C-onc
V-onc
MRNA W
MRNA
Abnormal protein
(hyperactive)
Protein
product
Abnormal
cell growth
Chapter 16 Solutions
Human Heredity: Principles and Issues (MindTap Course List)
Ch. 16.5 - Should fertility clinics be required to disclose...Ch. 16.5 - Prob. 2EGCh. 16 - Would ISCI be an option? Why or why not? Jan, a...Ch. 16 - Jan is concerned about using ART. She wants to be...Ch. 16 - List the common infertility problems in women....Ch. 16 - Prob. 2QPCh. 16 - Prob. 3QPCh. 16 - What is the difference between gamete...Ch. 16 - Why should women consider collecting and freezing...Ch. 16 - Prob. 6QP
Ch. 16 - Researchers are learning how to transfer...Ch. 16 - Prob. 8QPCh. 16 - Cystic fibrosis is an autosomal disease that...Ch. 16 - Prob. 10QPCh. 16 - Gene therapy involves: a. the introduction of...Ch. 16 - In selecting target cells to receive a transferred...Ch. 16 - The prospect of using gene therapy to alleviate...Ch. 16 - Is gene transfer a form of eugenics? Is it...Ch. 16 - A couple who wishes to have children visits you, a...Ch. 16 - A couple has had a child born with...Ch. 16 - You are a genetic counselor, and your patient has...Ch. 16 - Prob. 18QP
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- Explain how viral vectors can be used for gene therapy, and provide two examples of commonly used viral vectors. What are some of the major challenges that must be overcome to develop safer and more effective viral vectors for gene therapy?arrow_forwardCloning vectors are not just limited to bacterial plasmids. Bacteriophages and M13 phage vectors are also commonly utilized in the cloning process. State any five (5) key criteria to be an effective cloning vector.arrow_forwardWhich of the following conditions is most likely to be successfully treated by gene therapy using a viral vector to deliver a wild-type copy of one gene that is present in mutant form in a person with the condition? Assume that the viral vector used has the ability to home to relevant target tissues. Also assume that the patient can be treated at a young enough age to avoid irreversible phenotypic impacts of the mutation described. Timothy Syndrome, a multi-system disorder characterized by dysmorphic features and autistic behavioral traits, caused by overexpression of calcium channel gene Cerebral adrenoleukodystrophy (ALD), a neurological disorder caused by a frameshift mutation knocking out the function of the ABCD1 gene encoding a lipid transporter Osteogenesis imperfecta caused by a dominant negative mutation in the collagen A1 gene Myopia (shortsightedness), a vision impairment with heritability estimates in the range of 0.6-0.8, where risk is impacted by at least 200 genesarrow_forward
- Two approaches for correcting single-gene defects are gene therapy such as is discussed in Section 3-5D and the CRISPR–Cas9 system. Explain why the CRISPR–Cas9 approach can potentially provide more complete restoration of normal tissue function than gene therapy.arrow_forward1. You are part of a research group that wants to create a strain of humans resistant to COVID-19 by inserting the "Corona Virus Resistance Gene" into the next generation of humans. In order to achieve this, you must design primers that would amplify the "Corona Virus Resistance Gene." a) Using the diagram provided, describe where the forward and reverse primers would bind for amplification. b) Would this construct use the same primers as Dr. Devlin used in his Growth Hormone construct? Explain. Gene Gene Gene Terminator Promoter 2. a) If you started with 5 DNA strands and the above diagram repeated for 9 cycles, how th many DNA strands would you have after the 9" cycle? b) What would happen if you forgot to add dNTPs to the PCR reactions? Would any DNA amplification occur? 3. A linear DNA molecule is digested by the LoIRI restriction enzyme that cuts DNA at four different positions. After completing the restriction digest, you use gel electrophoresis to visualize the DNA fragments.…arrow_forwardExplain how site directed mutagenesis can be accomplished using M13 bacteriophage. Using this method often results in only 5% of plaques with mutant gene. Explain how the procedure has been modified to enrich for the number of plaques with the mutant gene.arrow_forward
- Some Covid 19 vaccines in development use the adenovirus, a common vector used in gene therapies, to inject the mRNa for the spike protein into human muscle cells. Why might vaccines using a virus vector be more likely to need only refrigeration and not deep-cold storage? Some older vaccines used a weakened strain of the pathogenic virus in the injection in the hope that the weakened virus would induce an immune response without causing disease. The virus vectors used in genetic engineering have all the harmful virus genes cut out and replaced with the foreign gene, such as one that makes a protein that is absent in the human patient. Which type of viral vaccine would you prefer to take as the safest option? Why?arrow_forwardA neutral mutation is, by definition, a mutation that does not result in the change of the encoded amino acid sequence of a gene. 1)True 2)Falsearrow_forwardWith the use of well-illustrated diagrams, reconstruct the entire cloning process by explaining different stages of the cloning process that involves the following: d. Screening/selection of hosts containing the intended recombinant plasmid. For this stage(d), discuss the importance of a second marker that can be used for screening of genomic DNA for colonies containing the pka-1 under the principle of insertional inactivation. This should be properly explained using all the attributes of the plasmid described above.arrow_forward
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