Essentials Of Genetics (9th Global Edition)
9th Edition
ISBN: 9780134143637
Author: William S. Klug, Michael R. Cummings
Publisher: Pearson Global Edition
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Chapter CHST6, Problem 6RQ
Summary Introduction
To review:
The case of Ashanti DeSilva who was treated for SCID using gene therapy. To know whether the gene delivered has replaced the defective copy of the ADA gene and analyze the reason for choosing white blood cells as the targets for the therapeutic gene.
Introduction:
The first approved human gene therapy trial was on a 4-year-old girl. This girl named Ashanti DeSilva was suffering from SCID. SCID stands for severe combined immunodeficiency, which is a very rare and inheritable disorder.
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During the first successful gene therapy trial in which Ashanti DeSilva was treated for SCID, did the therapeutic gene delivered to Ashanti replace the defective copy of the ADA gene? Why were white blood cells chosen as the targets for the therapeutic gene?
What percentage of cells in an organ or a tissue need toexpress a therapeutic gene to alleviate the effects of agenetic disorder?
In the plaque assay, what is the precise origin of a single plaque?
Chapter CHST6 Solutions
Essentials Of Genetics (9th Global Edition)
Ch. CHST6 - What is gene therapy?Ch. CHST6 - Prob. 2RQCh. CHST6 - Prob. 3RQCh. CHST6 - Prob. 4RQCh. CHST6 - Explain how viral vectors can be used for gene...Ch. CHST6 - Prob. 6RQCh. CHST6 - Explain an example of successful gene therapy...Ch. CHST6 - Prob. 8RQCh. CHST6 - Prob. 9RQCh. CHST6 - Describe two gene-silencing techniques and explain...
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- The acquired antibiotic resistance genes encode what types of proteins that can inhibit the function of the antibiotic? list twoarrow_forwardWith regard to the CRISPR-Cas system that defends bacteriaagainst bacteriophage attack, what happens during the adaptation,expression, and interference phases? When a bacterium is exposed toa particular bacteriophage, is the adaptation phase always necessary?arrow_forwardHow can we be sure that the insertion of the therapeutic gene does not harm some other necessary cell function?arrow_forward
- What does MEGA stand for and what is this assay used to determine? How to do perform the assay?arrow_forwardWhat mechanism of antibiotic resistance does the NDM gene codefor?arrow_forwardrs1800092 is a single nucleotide variant identifier for a variant in the CFTR gene. What type of variant is it? What is the amino acids change? Is it pathogenic?arrow_forward
- What is double-knockout animals (DKOs) ?arrow_forwardIn selecting target cells to receive a transferred gene in gene therapy, what factors do you think would have to be taken into account? Explain your answer.arrow_forwardwhich is the delivery techniques can bbe used to best transfer recombinant plasmids into E.coli cells?arrow_forward
- Do the antibiotic-resistant mutants arise in response to the presence of the antibiotic?arrow_forwardIf a vector containing the desired gene X is inserted into a microorganism, for instance, A.carbornarious, and the protoplast transformation resulted in 9 gene X transformants, how to determine which transformant has the highest titer of gene X?arrow_forwardTo understand the genes responsible for growth and infectivity in a disease-causing bacterial strain, you perform chemical mutagenesis on a culture of these bacteria. In the course of your investigation into the properties of the resulting mutants, you identify a set of mutant bacteria that is still viable but their virulence is significantly impaired. How might these mutants be useful for vaccine development?arrow_forward
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