Genetics: From Genes to Genomes
6th Edition
ISBN: 9781259700903
Author: Leland Hartwell Dr., Michael L. Goldberg Professor Dr., Janice Fischer, Leroy Hood Dr.
Publisher: McGraw-Hill Education
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Chapter 1, Problem 18P
Researchers have successfully used gene therapy to ameliorate some human genetic diseases by adding a normal gene copy to cells whose genomes originally had only nonfunctional mutant copies of that gene. For example, a form of blindness due to the lack of a single protein called RPE65 has been reversed by introduction of a normal RPE65 gene to cells of the retina of adults.
| a. | The success of this gene therapy approach provides us with clues about the role of the RPE65 protein in the retina. Do you think that RPE65 is needed for the proper development of the human eye? |
| b. | Can you see a potential difficulty in applying this gene therapy approach for diseases like microcephaly? |
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Researchers have successfully used gene therapy toameliorate some human genetic diseases by adding anormal gene copy to cells whose genomes originallyhad only nonfunctional mutant copies of that gene.For example, a form of blindness due to the lack of asingle protein called RPE65 has been reversed byintroduction of a normal RPE65 gene to cells of theretina of adults.a. The success of this gene therapy approach providesus with clues about the role of the RPE65 proteinin the retina. Do you think that RPE65 is neededfor the proper development of the human eye?b. Can you see a potential difficulty in applying this genetherapy approach for diseases like microcephaly?
What is the first step in quantifying the relative amounts of mRNA in different tissues? Would this method be useful in determining which immune system genes might be over-expressed in severe Covid cases? Why or why not?
Could quantitative PCR, which uses a DNA-binding dye, to show how many copies of the target DNA sequence could be used to quantify the amount of mRNA in a cell? Would you expect that a metabolically active tissue such as the liver would show more cDNA copies in such a method, compared to less metabolically active tissues such as skin cells?
One reason that the types and amounts of mRNAs are quantified in different tissue types is to compare which genes are activated and which are inactive. It used to be thought that any gene that was transcribed was automatically translated. The discovery of RNA-degrading systems shows that the real situation in cells is more complemented. Do you believe that a larger amount of mRNA of a given type, say for alpha hemoglobin in…
. In selecting target cells to receive a transferred gene in gene therapy in an adult person, what factors do you think would have to be taken into account for a successful treatment?
Chapter 1 Solutions
Genetics: From Genes to Genomes
Ch. 1 - Choose the phrase from the right column that best...Ch. 1 - If one strand of a DNA molecule has the base...Ch. 1 - The size of one copy of the human genome is...Ch. 1 - Indicate whether each of the following words or...Ch. 1 - a. How many different DNA strands composed of 100...Ch. 1 - RNA shares with proteins the ability to fold into...Ch. 1 - The human protein lactate dehydrogenase shown in...Ch. 1 - a. Are the triplets in the genetic code table...Ch. 1 - Why do scientists think that all forms of life on...Ch. 1 - Why would a geneticist study a yeast cell or a...
Ch. 1 - How can a scientist tell if a protein present in...Ch. 1 - Figure 1.6 shows the amino acid sequences of parts...Ch. 1 - Why do scientists think that new genes arise by...Ch. 1 - Explain how the exon/intron structure of genes...Ch. 1 - Mutations in genes that change their pattern of...Ch. 1 - A single zebrafish gene function was inactivated...Ch. 1 - Different mutations in the WDR62 gene that...Ch. 1 - Researchers have successfully used gene therapy to...Ch. 1 - By the time this book is published, it will likely...
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- Although it is well known that X-rays cause mutations, they are routinely used to diagnose medical problems, including potential tumors, broken bones, and dental cavities. Why is this done? What precautions need to be taken?arrow_forwardDescribe the mechanistic steps through which gene therapy might be used to cure a disease that is caused by mutations in "Gene D". A potential patient is reluctant to undergo this treatment, because they remember hearing about adverse incidents from gene therapy studies conducted decades ago. What relevant details might you tell them, in order to inform them on this topic?.arrow_forwardCould quantitative PCR, which uses a DNA-binding dye, to show how many copies of the target DNA sequence could be used to quantify the amount of mRNA in a cell? Would you expect that a metabolically active tissue such as the liver would show more cDNA copies in such a method, compared to less metabolically active tissues such as skin cells? One reason that the types and amounts of mRNAs are quantified in different tissue types is to compare which genes are activated and which are inactive. It used to be thought that any gene that was transcribed was automatically translated. The discovery of RNA-degrading systems shows that the real situation in cells is more complemented. Do you believe that a larger amount of mRNA of a given type, say for alpha hemoglobin in immature red blood cells is a reliable indicator that more alpha hemoglobin protein will be made in those cells?arrow_forward
- Gene expression can be disrupted by techniques such as homologous recombination and RNA interference. What is the functional difference between these two methods in terms of the ultimate effect on gene expression? Why might homologous recombination or an alternative genome-editing method (e.g., CRISPR/Cas9) be preferred over RNA interference?arrow_forwardIn the bacteriophage T7 system used to express recombinant proteins, the gene of interest is fused to T7 promoter and T7 RNA polymerase is separately cloned into the same cell. What is the main reason this system uses T7 RNA polymerase instead of relying on the bacterial RNA polymerase? To restrict the expression of bacterial protein expression To enhance the amount of recombinant protein expression To enhance the expression of bacterial protein expression To restrict the amount of recombinant protein expression To enable the expression of T7 viral protein expressionarrow_forwarda. Some antibiotics, such as rifampin, interfere with the function of RNA polymerase. What biological process is rifampin disrupting? b. Some antibiotic-resistant M. tuberculosis bacteria have a single point mutation (CàT) in the rpoB gene that causes an amino acid change from serine (a polar amino acid) to leucine (a non-polar amino acid). What type of mutation is this? Do you expect this to have no effect, a small effect, or a large effect on the polypeptide produced? Explain your reasoning. c. The rpoB gene encodes a subunit of the bacterial RNA polymerase protein. The point mutation described in Question 2 causes a change in protein folding, which leads to the inability of the rifampin antibiotic to bind to the RNA polymerase. Which level(s) of protein structure is/are affected by this change?arrow_forward
- Sickle cell disease is caused by a substitution in the beta globin gene. As a result, one amino acid is different in the mutant protein. Which of the following region is likely to be affected in the sickle cell allele? A. The promoter region B. The 5' UTR region C. The coding region D. The 3' UTRarrow_forwardIf a mutation occurred in a control element sequence associated with a particular gene, which of the following would be a likely consequence of this? A. This mutation is not likely to affect the expression of the associated gene, because RNA polymerase does not bind to control elements. B. This will likely affect the expression of the associated gene because it will loosen the coiling of chromatin at this site. C. The expression of the associated gene will be affected because this change will result in a nonsense mutation. D. This mutation is not likely to affect the expression of the associated gene, since control elements are noncoding sequences. E. The associated gene's expression would likely be affected, since this mutation would change the ability of at least one transcription factor to bind.arrow_forwardConsider a single base insertion mutation between the 3rd and 4th codons in a natural gene that encodes a protein 100 amino acids long. Is it more likely that the protein produced by this mutant allele will be shorter or longer than 100 amino acids long?arrow_forward
- Which of the following is an explanation for how there can be differential gene expression throughout a multicellular organism? A) There are unique sets of genes present in the different tissues of the organisms B) Some tissues have histones, while other cells do not use histones C) There are unique combinations of transcription factors in different tissues of the organism D) Different tissues throughout the organism use different genetic coding rules .arrow_forwardSuppose researchers learn that a particular congenital disease is caused by synthesis of a protein coded by a mutated gene. Describe a way in which recombinant DNA technology might be used to prevent translation of the protein.arrow_forwardWhich of the following mutations would have the greatest negative impact on the protein product of a gene? A. a single base deletion close to the end of the coding region of a gene. B. a single base insertion near the start of the coding region of the gene C. a base-pair substitution D. a deletion of three bases near the middle of the genearrow_forward
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