Question 1

With regard to treatment of inborn errors of metabolism (IEM), which of the following statements, if any, is false?

Accepted Answer

A)  IEMs are all single gene disorders that have been studied for many decades, leading to the development of successful treatment in all cases. 
B)  IEMs can be treated by augmentation therapy, treatment to inhibit positively harmful effects, or by prevention therapy. 
C)  Treatment for some individual IEMs can involve both augmentation therapy plus treatment to inhibit positively harmful effects. 
D)  Treatment of some IEMs involves artificially forcing an increase in a minor metabolic pathway to counteract a build-up in a toxic metabolite produced by a metabolic block in a major metabolic pathway. 
A)  IEMs are all single gene disorders that have been studied for many decades, leading to the development of successful treatment in all cases. 
B)  IEMs can be treated by augmentation therapy, treatment to inhibit positively harmful effects, or by prevention therapy. 
C)  Treatment for some individual IEMs can involve both augmentation therapy plus treatment to inhibit positively harmful effects. 
D)  Treatment of some IEMs involves artificially forcing an increase in a minor metabolic pathway to counteract a build-up in a toxic metabolite produced by a metabolic block in a major metabolic pathway.

Question 2

Many of the genes that produce the enzymes and other proteins involved in handling drugs are polymorphic. Why should that be?

Accepted Answer

From the body's perspective, drug metabo

Question 3

Sometimes, a prescribed drug can be dangerous, and occasionally deadly, according to a patient's genotype. Give three examples of such.

Accepted Answer

1) Suxamethonium (succinylcholine) works

Question 4

Which, if any, of the following statements is false?

Accepted Answer

A)  RNA interference (RNAi) is a cellular defense mechanism that is triggered by the presence in cells of unnatural double-stranded RNA, as can occur after viral infections. 
B)  RNAi therapy is a type of RNA-targeted therapy in which specific double-stranded RNA constructs are engineered to appear in diseased cells in order to incite the cells to destroy any RNA that contains the same sequence. 
C)  By destroying RNAs that are related to a specifically introduced genetic construct, artificial RNAi is effectively a type of gene-specific silencing. 
D)  RNAi therapy is best suited to silencing genes so as to replicate a phenotype caused by loss-of-function mutations. 
A)  RNA interference (RNAi) is a cellular defense mechanism that is triggered by the presence in cells of unnatural double-stranded RNA, as can occur after viral infections. 
B)  RNAi therapy is a type of RNA-targeted therapy in which specific double-stranded RNA constructs are engineered to appear in diseased cells in order to incite the cells to destroy any RNA that contains the same sequence. 
C)  By destroying RNAs that are related to a specifically introduced genetic construct, artificial RNAi is effectively a type of gene-specific silencing. 
D)  RNAi therapy is best suited to silencing genes so as to replicate a phenotype caused by loss-of-function mutations.

Question 5

Fill in the missing blanks with single words.
Genome  ______ means artificially introducing a specific change in the DNA sequence at a unique, pre-determined location within the genome of an ____2____ cell. The method relies on some form of recognition of specific sequences on both DNA strands at a locus that then allows an artificially introduced _____3____-stranded DNA break at this location. In response to the _____3____-stranded DNA break, DNA repair is carried out by the cell but after using non_____4_____ end joining DNA repair, errors can be made in the repair that can occasionally result in a desired specific DNA change. In one system genetically engineered ______5_____ ______6_____ nucleases are used in which a DNA is constructed to code for a specific sequence of ______5______ _____6______ and is then ligated to a DNA sequence that will specify a DNA ______7______ domain. A plasmid containing the resulting DNA construct can encode a ______5______ _____6______ nuclease when transfected into a cell. Using this technology, a pair of ______5______ _____6______ nucleases can be designed to bind to specific sequences on the opposite DNA strands at a desired unique position in the genome and the adjoining DNA cleavage domains work to produce the required ____3_____-strand DNA break.

Accepted Answer

1. editing. 
2. inta

Question 6

Which, if any, of the following statements is false?

Accepted Answer

A)  Transdifferentiation means reprogramming of a differentiated cell so that it acquires the characteristics of another type of differentiated cell. 
B)  In dedifferentiation a differentiated cell is artificially reprogrammed so that it behaves as a pluripotent cell. 
C)  Human induced pluripotent stem (iPS) cell lines are usually generated by artificial dedifferentiation of readily accessible human cells, such as skin cells. 
D)  Human iPS cell technologies do not offer clinical applications but they are of value for studying pathways of cellular differentiation 
A)  Transdifferentiation means reprogramming of a differentiated cell so that it acquires the characteristics of another type of differentiated cell. 
B)  In dedifferentiation a differentiated cell is artificially reprogrammed so that it behaves as a pluripotent cell. 
C)  Human induced pluripotent stem (iPS) cell lines are usually generated by artificial dedifferentiation of readily accessible human cells, such as skin cells. 
D)  Human iPS cell technologies do not offer clinical applications but they are of value for studying pathways of cellular differentiation

Question 7

In some cases, genetic variation at multiple loci is known to affect the response to a specific drug. What is known about genetic variation that affects our responses to the anticoagulant warfarin?

Accepted Answer

At least three loci are involved: CYP2C9

Question 8

How has exon skipping therapy been applied to treat Duchenne muscular dystrophy?

Accepted Answer

Internal deletions are a common cause of

Question 9

Fill in the missing blanks with single words.
In the recent past, virus vectors used in gene therapy trials were often based on a type of retrovirus called a  _______retrovirus. They had the advantage of allowing a _____2_____ DNA to be stably inserted into the _____3____ DNA of cells. For cells that are short-lived, such as blood cells, the hope was that a certain percentage of _____4_____ cells might be successfully transduced so that there was a self-renewing population of cells carrying the desired _____2_____ DNA. Unfortunately, vectors based on  _______retroviruses have a poor safety profile: there is little control over where they ______5_______ into the _____3____ DNA and sometimes when they _____5_____ they activate a neighboring _____6_____, causing _____7_____. As a result, in modern gene therapy trials it is now commonplace to use self-_____8______ strains of a class of retrovirus vectors known as _____9____ that are much safer to use.

Accepted Answer

1. gamma. 
2. therapeutic. 
3.

Question 10

Concerning making animal models of human disease, which, if any, of the following statements is false?

Accepted Answer

A)  Pronuclear microinjection is a general way of making a transgenic animal and involves microinjection of foreign DNA into a fertilized egg cell. 
B)  Pronuclear microinjection is best suited to modelling recessively inherited single gene disorders. 
C)  Gene targeting using embryonic stem cells depends on having well-characterized embryonic stem cell lines that can readily allow transmission through the germ line. 
D)  Gene targeting using embryonic stem cells in mice is a popular way of modelling disease phenotypes that result from a gain-of-function. 
A)  Pronuclear microinjection is a general way of making a transgenic animal and involves microinjection of foreign DNA into a fertilized egg cell. 
B)  Pronuclear microinjection is best suited to modelling recessively inherited single gene disorders. 
C)  Gene targeting using embryonic stem cells depends on having well-characterized embryonic stem cell lines that can readily allow transmission through the germ line. 
D)  Gene targeting using embryonic stem cells in mice is a popular way of modelling disease phenotypes that result from a gain-of-function.

Question 11

How might a form of germ-line gene therapy be used to treat severe mitochondrial diseases?

Accepted Answer

Mutations in mitochondrial DNA (mtDNA) a

Question 12

Which of the following statements, if any, is false?

Accepted Answer

A)  Monoclonal antibodies are made by identical immune cells and so will recognize and bind just one specific epitope on a target molecule. 
B)  Monoclonal antibodies of rodent origin are far from ideal therapeutic agents because of their short half-life in human serum and the potential for immune responses by the recipient. 
C)  Humanized antibodies are hybrid antibodies that have constant regions of human origin but variable regions of rodent origin. 
D)  An intrabody is an artificial constructs with just a single chain that is linked to variable domains and, unlike regular antibodies with four polypeptide chains, has the potential to work inside cells. 
A)  Monoclonal antibodies are made by identical immune cells and so will recognize and bind just one specific epitope on a target molecule. 
B)  Monoclonal antibodies of rodent origin are far from ideal therapeutic agents because of their short half-life in human serum and the potential for immune responses by the recipient. 
C)  Humanized antibodies are hybrid antibodies that have constant regions of human origin but variable regions of rodent origin. 
D)  An intrabody is an artificial constructs with just a single chain that is linked to variable domains and, unlike regular antibodies with four polypeptide chains, has the potential to work inside cells.

Question 13

Which, if any, of the following can be classified as a type of augmentation therapy?

Accepted Answer

A)  Treatment using a small molecule drug to bind a target protein and prevent it working. 
B)  A bone marrow transplant. 
C)  Corrective surgery for cleft lip and palate. 
D)  Insulin treatment in diabetes. 
A)  Treatment using a small molecule drug to bind a target protein and prevent it working. 
B)  A bone marrow transplant. 
C)  Corrective surgery for cleft lip and palate. 
D)  Insulin treatment in diabetes.

Question 14

Describe the characteristics of two viral vectors based on RNA genomes and two viral vectors based on DNA genomes that are used in gene therapy.

Accepted Answer

-Gammaretroviruses. These are simple, si

Question 15

In mammals, pluripotent cells occur naturally in the early embryo but pluripotent cell lines can also be artificially created. The first approach was to make embryonic stem cell lines from cells isolated from early embryos. More recently, pluripotent cell lines have been made by artificially changing the epigenetic settings of differentiated cells. What is involved in the latter case?

Accepted Answer

Artificial epigenetic reprogramming can

Question 16

Which, if any, of the following statements is false?

Accepted Answer

A)  The great majority of clinical gene therapy trials have had limited success 
B)  The only successful gene therapies have been for recessive blood disorders. 
C)  The only successful gene therapies have been ex vivo gene therapies. 
D)  Gene therapy for inherited disorders represents a minority of clinical gene therapy trials. 
A)  The great majority of clinical gene therapy trials have had limited success 
B)  The only successful gene therapies have been for recessive blood disorders. 
C)  The only successful gene therapies have been ex vivo gene therapies. 
D)  Gene therapy for inherited disorders represents a minority of clinical gene therapy trials.

Question 17

Which, if any, of the following descriptions is false?

Accepted Answer

A)  Zinc fingers are elements of protein secondary structure in which the polypeptide chain folds back upon itself after co-ordination of a Zn2+ ion with selected amino acids, often a pair of cysteines and a pair of histidines. 
B)  Zinc finger nucleases are natural proteins containing a sequence of zinc fingers that can bind to specific sequences in DNA. 
C)  After zinc finger nucleases bind to both DNA strands at a specific DNA sequence they attract cellular DNA cleavage enzymes, inducing them to make a double-stranded break at just that one position in the genome. 
D)  The CRISPR-Cas system also allows genome editing but in this case the target DNA sequences are recognized by guide RNA sequences rather than proteins. 
A)  Zinc fingers are elements of protein secondary structure in which the polypeptide chain folds back upon itself after co-ordination of a Zn2+ ion with selected amino acids, often a pair of cysteines and a pair of histidines. 
B)  Zinc finger nucleases are natural proteins containing a sequence of zinc fingers that can bind to specific sequences in DNA. 
C)  After zinc finger nucleases bind to both DNA strands at a specific DNA sequence they attract cellular DNA cleavage enzymes, inducing them to make a double-stranded break at just that one position in the genome. 
D)  The CRISPR-Cas system also allows genome editing but in this case the target DNA sequences are recognized by guide RNA sequences rather than proteins.

Question 18

Four stages are often identified in drug development: a preclinical stage plus three clinical trial stages. What is involved in these?

Accepted Answer

-The pre-clinical stage involves a batte

Question 19

There are two broad principles regarding the technological aim of somatic gene therapy: (a) genetically modifying disease cells (without killing them), and (b) killing disease cells either directly or indirectly. Explain what is involved in the two strategies.

Accepted Answer

1) The idea is to alter gene expression

Question 20

Molecular therapeutic strategies sometimes target RNA instead of DNA. What is involved in RNA interference therapy, and how useful has it been?

Accepted Answer

RNA interference therapy is a type of ar

With regard to treatment of inborn errors of metabolism (IEM), which of the following statements, if any, is false?

Many of the genes that produce the enzymes and other proteins involved in handling drugs are polymorphic. Why should that be?

Sometimes, a prescribed drug can be dangerous, and occasionally deadly, according to a patient's genotype. Give three examples of such.

Which, if any, of the following statements is false?

Which, if any, of the following statements is false?

In some cases, genetic variation at multiple loci is known to affect the response to a specific drug. What is known about genetic variation that affects our responses to the anticoagulant warfarin?

How has exon skipping therapy been applied to treat Duchenne muscular dystrophy?

Concerning making animal models of human disease, which, if any, of the following statements is false?

How might a form of germ-line gene therapy be used to treat severe mitochondrial diseases?

Which of the following statements, if any, is false?

Which, if any, of the following can be classified as a type of augmentation therapy?

Describe the characteristics of two viral vectors based on RNA genomes and two viral vectors based on DNA genomes that are used in gene therapy.

Which, if any, of the following statements is false?

Which, if any, of the following descriptions is false?

Four stages are often identified in drug development: a preclinical stage plus three clinical trial stages. What is involved in these?

There are two broad principles regarding the technological aim of somatic gene therapy: (a) genetically modifying disease cells (without killing them), and (b) killing disease cells either directly or indirectly. Explain what is involved in the two strategies.

Molecular therapeutic strategies sometimes target RNA instead of DNA. What is involved in RNA interference therapy, and how useful has it been?

Fundamentals of Dna, Chromosomes, and Cells

Fundamentals of Gene Structure, Gene Expression, and Human Genome Organization

Principles Underlying Core Dna Technologies

Principles of Genetic Variation

Single-Gene Disorders: Inheritance Patterns, Phenotype Variability, and Allele Frequencies

Principles of Gene Regulation and Epigenetics

Genetic Variation Producing Diseasecausing Abnormalities in Dna and Chromosomes

Identifying Disease Genes and Genetic Susceptibility to Complex Disease

Cancer Genetics and Genomics

Genetic Testing From Genes to Genomes, and the Ethics of Genetic Testing and Therapy

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Exam 9: Genetic Approaches to Treating Disease

With regard to treatment of inborn errors of metabolism (IEM), which of the following statements, if any, is false?

Many of the genes that produce the enzymes and other proteins involved in handling drugs are polymorphic. Why should that be?

Sometimes, a prescribed drug can be dangerous, and occasionally deadly, according to a patient's genotype. Give three examples of such.

Which, if any, of the following statements is false?

Which, if any, of the following statements is false?

In some cases, genetic variation at multiple loci is known to affect the response to a specific drug. What is known about genetic variation that affects our responses to the anticoagulant warfarin?

How has exon skipping therapy been applied to treat Duchenne muscular dystrophy?

Concerning making animal models of human disease, which, if any, of the following statements is false?

How might a form of germ-line gene therapy be used to treat severe mitochondrial diseases?

Which of the following statements, if any, is false?

Which, if any, of the following can be classified as a type of augmentation therapy?

Describe the characteristics of two viral vectors based on RNA genomes and two viral vectors based on DNA genomes that are used in gene therapy.

Which, if any, of the following statements is false?

Which, if any, of the following descriptions is false?

Four stages are often identified in drug development: a preclinical stage plus three clinical trial stages. What is involved in these?

There are two broad principles regarding the technological aim of somatic gene therapy: (a) genetically modifying disease cells (without killing them), and (b) killing disease cells either directly or indirectly. Explain what is involved in the two strategies.

Molecular therapeutic strategies sometimes target RNA instead of DNA. What is involved in RNA interference therapy, and how useful has it been?

Fundamentals of Dna, Chromosomes, and Cells

Fundamentals of Gene Structure, Gene Expression, and Human Genome Organization

Principles Underlying Core Dna Technologies

Principles of Genetic Variation

Single-Gene Disorders: Inheritance Patterns, Phenotype Variability, and Allele Frequencies

Principles of Gene Regulation and Epigenetics

Genetic Variation Producing Diseasecausing Abnormalities in Dna and Chromosomes

Identifying Disease Genes and Genetic Susceptibility to Complex Disease

Cancer Genetics and Genomics

Genetic Testing From Genes to Genomes, and the Ethics of Genetic Testing and Therapy

Filters