Study Questions

1. Severe combined immunodeficiency (SCID) syndromes are excellent models for gene therapy because of the genetic basis of these disorders and significant advances in the technology to transfer therapeutic genes into hematopoietic precursor cells. For all these reasons, which of the following syndromes represents an ideal candidate for gene therapy?

(A) B-cell deficiency

(B) DiGeorge's syndrome

(C) 7C Deficiency

(D) Adenine deaminase deficiency

(E) T-cell deficiency

2. All of the following are desirable characteristics in the design of a gene therapy vector EXCEPT

(A) Ability to produce at high titer on a commercial scale

(B) Ability to transfect both dividing and nondi-viding cells

(C) Ability to produce site-specific integration into the chromosome of the target cell

(D) Ability to limit size of genetic material it can deliver

(E) Ability to deliver only certain cell types

3. A patient with ornithine transcarbamylase (OTC) deficiency is being treated in a gene therapy clinical trial. The gene therapy approach for this disease is primarily designed to

(A) Replace the enzyme ornithine transcarbamylase

(B) Decrease the accumulation of ammonia

(C) Eliminate the need for a modified diet

(D) Target a protooncogene

(E) Enhance the immune system

4. A 25-year-old hemophiliac is interested in receiving gene therapy. He should contemplate all of the following approaches EXCEPT

(A) Intravenous infusion of a retroviral vector expressing the B-domain-deleted factor VIII

(B) Ex vivo transfection of autologous fibroblasts transfected with a plasmid encoding B-domain-deleted factor VIII

(C) Intravenous adenoviral-mediated delivery of factor VIII

(D) Adeno-associated virus (AAV) vector delivered to skeletal muscle

(E) Retroviral vector expressing B-domain deleted factor VIII transfected into dermal fibroblasts that are then reimplanted

5. A patient with advanced inoperable squamous cell carcinoma of the head and neck receives a replication-selective adenovirus on a gene therapy clinical trial. The rationale for the use of this treatment:

(A) Deletion of viral genes will reduce toxicity of the viral vector to normal cells.

(B) Deletion of a p53 inhibitory protein will be selective for tumors that have lost p53 function.

(C) Deletion of a key regulatory sequence will allow for induction of the therapeutic gene in tumor cells.

(D) Results of preclinical studies suggest that only tumor cells are affected by this treatment.

(E) Clinical results support that only patients with p53 mutations in their tumors respond to the treatment.

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