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Which mode of gene therapy accomplishes transgene integration into the target cell's genome?


A) adenovirus-mediated
B) retrovirus-mediated
C) liposome-mediated
D) A and B

E) A) and B)
F) None of the above

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If DNA damage occurs early in G1 of the cell cycle, the tumor suppressor protein p53


A) activates the p21 gene which encodes a CDK inhibitor, thereby causing G1 arrest.
B) promotes apoptosis.
C) phosphorylates the retinoblastoma protein (pRB) , thereby blocking the cell cycle.
D) is targeted for degradation by the proteasome.

E) B) and C)
F) A) and D)

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You suspect that a gene you have cloned is a proto-oncogene. Describe how you would test your hypothesis experimentally. Predict the results.

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If the gene is a proto-oncogene then its...

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Can surgery successfully cure a patient of a cancer that has metastasized?


A) No; all the body cells of the patient are dividing uncontrollably.
B) Yes; surgery could remove all the patient's cells with defective cell-cycle regulation.
C) No; cancer cells are no longer localized in one spot in the patient.
D) Yes; if the patient's tumor is benign.

E) A) and D)
F) None of the above

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Diagram the hypothesized action of a hairpin-derived catalytic antisense RNA against the HIV-1 long terminal repeat (LTR).

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To diagram the hypothesized action of a ...

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Compare and contrast the strategies used for the treatment of ornithine transcarbamylase (OTC) deficiency, adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID), and cystic fibrosis. Why are different treatment strategies and different vectors used for different genetic diseases?

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Ornithine transcarbamylase (OTC) deficie...

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Currently, somatic cell gene therapy:


A) has only been tested in cell culture and animal models
B) is experimental and has never achieved any clinical success.
C) is experimental and has achieved only very limited clinical success.
D) is routine and has been used successfully to treat many diseases.

E) C) and D)
F) A) and B)

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Diagram the two main mechanisms for transformation of cells by retrovirus infection.

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The two main mechanisms for transformati...

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What is an oncogene?


A) A gene associated with tumor formation when its product does not function.
B) A gene associated with tumor formation when it is inappropriately activated.
C) A gene that inhibits cell growth when its product functions normally.
D) A gene that codes for a transcription factor involved in tumor formation.

E) None of the above
F) B) and D)

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Draw a diagram illustrating how inappropriate activation of proto-oncogenes may be due to qualitative or quantitative changes.

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Inappropriate activation of proto-oncoge...

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Explain why c-myc is referred to as a central "oncogenic switch."

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C-myc is referred to as a central "oncog...

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To create a "safe" viral vector for delivery of genes into human cells, the virus must be engineered to:


A) remove all viral coat proteins.
B) remove all viral genes, replacing them with the human genes to be delivered.
C) remove select viral genes and replace them with the human genes to be delivered.
D) remove the viral genome and coat proteins and replace them with plasmids carrying the human genes to be delivered.

E) All of the above
F) B) and C)

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The process by which cancer cells travel from the tissue of origin to other parts of the body is called


A) immortalization
B) transformation
C) metastasis
D) carcinogenesis

E) A) and B)
F) B) and D)

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Explain the molecular mechanism for control of chronic myelogenous leukemia by imatinib mesylate (Gleevac).

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Chronic myelogenous leukemia (CML) is a ...

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The "Philadelphia chromosome" is a reciprocal translocation of chromosome 9 to 22, which causes chronic myelogenous leukemia. The chromosomal translocation fuses the genes encoding the tyrosine kinase ABL and BCR, an activator of ABL. The drug Gleevac has been shown to be effective in Phase II clinical trials. The drug works by:


A) dephosphorylating ABL substrates
B) blocking the active site of the ABL tyrosine kinase so that ATP cannot bind
C) binding to BCR in a way that inhibits its ability to activate ABL
D) degrading the ABL-BCR fusion protein

E) None of the above
F) All of the above

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Explain how deletions of both alleles of p53, or missense point mutations in one allele, can each lead to unrestrained cell growth.

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Deletions of both alleles of p53 or miss...

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Retrovirus vectors for gene therapy have all of the following characteristics except:


A) they can infect both dividing and nondividing cells.
B) they have a limited insert size.
C) they have generate only a mild immune response
D) they stably integrate into host chromosomes

E) A) and B)
F) A) and C)

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Which class of diseases is least amenable to treatment by gene therapy?


A) monogenic loss-of-function metabolic diseases
B) multigenic behavioral diseases
C) viral diseases
D) immunodeficiencies

E) C) and D)
F) B) and C)

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Cancer is a multi-step disease that requires


A) at least four to eight genetic changes over the course of years
B) mutation of the cancer gene
C) at least 20 genetic changes over the course of years
D) at least four to eight genetic changes over the course of days

E) A) and C)
F) None of the above

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Compare and contrast in vivo and ex vivo somatic cell gene therapy approaches.

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In vivo and ex vivo somatic cell gene th...

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