Concepts of Genetics (12th Edition)
12th Edition
ISBN: 9780134604718
Author: William S. Klug, Michael R. Cummings, Charlotte A. Spencer, Michael A. Palladino, Darrell Killian
Publisher: PEARSON
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Textbook Question
Chapter ST.5, Problem 7RQ
Explain an example of a successful gene therapy trial. In your answer be sure to consider: a description of the disease condition that was treated, the mutation or disease gene affected, the therapeutic gene delivered, and the method of delivery use for the therapy.
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Answer the two parts of the question.
a) Explain what gene therapy involves.b) Discuss how gene therapy can be used to treat/cure or prevent a disease and include in your answer any current challenges associated with its use.
Discuss the advantages and disadvantages of gene therapy. A limitedamount of funding is available for gene therapy research. Makea priority list of the three top diseases for which you would fundresearch. Discuss your choices.
List three conditions for which gene therapy trials have been performed for, and suggest reasons for their success
Chapter ST Solutions
Concepts of Genetics (12th Edition)
Ch. ST.1 - What is the difference between innate immunity and...Ch. ST.1 - What evidence demonstrates that CRISPR-Cas is an...Ch. ST.1 - Prob. 3RQCh. ST.1 - Why was the type II CRISPR-Cas9 system of S....Ch. ST.1 - Prob. 5RQCh. ST.1 - What is a single guide RNA, and what role does it...Ch. ST.1 - What is the difference between nonhomologous...Ch. ST.1 - Prob. 8RQCh. ST.1 - Prob. 9RQCh. ST.1 - Prob. 1DQ
Ch. ST.1 - Prob. 2DQCh. ST.1 - What ethical and safety considerations must be...Ch. ST.1 - Recall (from Chapter 18) how miRNAs and the...Ch. ST.1 - Describe two different ways in which engineered...Ch. ST.1 - Consider the following human genetic diseases:...Ch. ST.1 - What are the different concerns about off-target...Ch. ST.2 - What is VNTR profiling, and what are the...Ch. ST.2 - Prob. 2RQCh. ST.2 - Describe capillary electrophoresis. How does this...Ch. ST.2 - What are the advantages and limitations of...Ch. ST.2 - Prob. 5RQCh. ST.2 - Explain why mitochondrial DNA profiling is often...Ch. ST.2 - Prob. 7RQCh. ST.2 - Describe the database system known as CODIS. What...Ch. ST.2 - Prob. 9RQCh. ST.2 - Prob. 10RQCh. ST.2 - Given the possibility that synthetic DNA could be...Ch. ST.2 - Prob. 2DQCh. ST.2 - If you were acting as a defense lawyer in a murder...Ch. ST.2 - The phenomena of somatic mosaicism and chimerism...Ch. ST.3 - What is pharmacogenomics, and how does it differ...Ch. ST.3 - Describe how the drug Herceptin works. What types...Ch. ST.3 - Prob. 3RQCh. ST.3 - Prob. 4RQCh. ST.3 - Prob. 5RQCh. ST.3 - Prob. 6RQCh. ST.3 - Why is it necessary to examine gene-expression...Ch. ST.3 - Prob. 8RQCh. ST.3 - Prob. 1DQCh. ST.3 - Prob. 2DQCh. ST.3 - How can we ensure that a patients privacy is...Ch. ST.3 - As gene tests and genomic sequences become more...Ch. ST.4 - How do genetically modified organisms compare with...Ch. ST.4 - Prob. 2RQCh. ST.4 - Prob. 3RQCh. ST.4 - Prob. 4RQCh. ST.4 - Describe the mechanisms by which the Cry proteins...Ch. ST.4 - Prob. 6RQCh. ST.4 - Prob. 7RQCh. ST.4 - Describe how plants can be transformed using...Ch. ST.4 - How do positive and negative selection techniques...Ch. ST.4 - Prob. 10RQCh. ST.4 - What are the laws regulating the development,...Ch. ST.4 - Do you think that foods containing GM ingredients...Ch. ST.4 - Prob. 3DQCh. ST.5 - What is gene therapy?Ch. ST.5 - Prob. 2RQCh. ST.5 - When treating a person by gene therapy, is it...Ch. ST.5 - Describe two ways that therapeutic genes can be...Ch. ST.5 - Explain how viral vectors can be used for gene...Ch. ST.5 - Prob. 6RQCh. ST.5 - Explain an example of a successful gene therapy...Ch. ST.5 - Prob. 8RQCh. ST.5 - Prob. 9RQCh. ST.5 - Prob. 10RQCh. ST.5 - Prob. 11RQCh. ST.5 - Prob. 1DQCh. ST.5 - Who should be treated by gene therapy? What...Ch. ST.5 - The lifetime costs for treatment of conditions...Ch. ST.5 - Should CRISPR-Cas or other techniques be used for...Ch. ST.5 - Prob. 5DQCh. ST.6 - What are RFLP markers and how were they used to...Ch. ST.6 - Why was information from Nancy Wexlers large...Ch. ST.6 - How do aggregates of mHTT protein form?Ch. ST.6 - Why are the results from the inducible mouse model...Ch. ST.6 - Based on the results from mouse models, is it...Ch. ST.6 - What do the results from creating transgenic mice...Ch. ST.6 - What steps lead from the binding of the mHTT...Ch. ST.6 - Summarize the approaches to therapy designed to...Ch. ST.6 - There are nine known progressive neurodegenerative...Ch. ST.6 - Prob. 2DQCh. ST.6 - Prob. 3DQCh. ST.6 - Why is there an inverse correlation between the...Ch. ST.6 - Discuss the ethical issues raised by the use a...
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- Discuss the Genetically Modified Organism (Benefits and Risks)arrow_forwardWhat do you mean by treatment of genetic disorders using gene therapy? Please answer at your own words, please.arrow_forwardIf human genetics is leading toward preventive medicine, give the most significant expectations regarding gene therapy (please explain in detail).arrow_forward
- What do you understand by treatment of genetic disorders using gene therapy? Please answer at your own words, please.arrow_forwardDiscuss potential side effects of gene therapy.arrow_forwardSelect the correct statement/s regarding the application of biotechnology in the field of health and medicine. The clones with mutated genes develop on the photographic film due to the complementarity of the probe with the mutated gene in auto- radiography. Preservatives, stabilizers, and adjuvants are added to recombinant DNA vaccines in order to allow the usage of multi-dosage vials, enhancement in storage life, and immune response. If ADA deficiency is detected in the early embryonic stage, then it can be permanently cured by introducing a functional ADA gene into the Question Option A Option B Option C bone marrow cells. Both (B) and (C) Option D Provide Correct Option Herearrow_forward
- Select the correct statement/s regarding the application of biotechnology in the field of health and medicine. The clones with mutated genes develop on the photographic film due to the complementarity of the probe with the mutated gene in auto-radiography. Preservatives, stabilizers, and adjuvants are added to recombinant DNA vaccines in order to allow the usage of multi-dosage vials, enhancement in storage life, and immune response. If ADA deficiency is detected in the early embryonic stage, then it can be permanently cured by introducing a functional ADA gene into the bone marrow cells. Both (B) and (C)arrow_forwardA patient is diagnosed with lung cancer using imaging. To establish the best treatment option, doctors ask to profile the gene expression of the tumor, using a biopsy and microarray analysis. Describe the steps involved in the analysis in detail. The analysis reveals that a new gene therapy could be beneficial to the patient. Explain briefly how gene therapy could be used to cure cancer and detail how the treatment can be formulated for delivery to the patient. To prepare DNA for the gene therapy formulation, it is often important to measure the size of the molecule. Explain how this measurement can be done using agarose gel electrophoresis, taking care to detail the property of the DNA molecule at the basis of the technique. To generate large amounts of DNA to manufacture gene therapy payloads or to be able to see them on gel electrophoresis, specific sequences can be amplified by PCR. For a sequence of a 100 base pairs, calculate the number cycles of PCR required to generate 1 ng of…arrow_forwardWhat is the importance of gene silencing in hereditary disease. What are the application of gene therapy in the field of Medical Technology.arrow_forward
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