Gene Therapy

Question 1

Which one of the following is usually NOT part of a vector for gene therapy?
A. Coding sequence
B. Homologous sequence
C. Insulator sequences
D. Origin of DNA replication
E. Regulatory sequence

Answer 1

D. Origin of DNA replication

Question 2

Which one of the following gene therapy approaches would be best suited for using a non-viral plasmid-based vector?
A. Anti-sense RNA therapy
B. Correction of protein folding problem
C. Delivery of a genetic vaccine
D. Long-term production of a gene product
E. Suppression of an activating variant in a target cell its mitotic offspring

Answer 2

C. Delivery of a genetic vaccine

If sustained expression is not required, a simple plasmid vector may be sufficient to give a burst of gene expression activity to achieve the goal. This is particularly true when achieving cell death or providing a genetic vaccine is the intended goal of the gene transfer.

Question 3

Increasing the expression of fetal hemoglobin with butyrate in a patient with sickle cell disease can be considered as a form of
A. epigenetic therapy
B. gene editing therapy
C. germ line gene therapy
D. RNA modulation therapy
E. suppression gene therapy

Answer 3

A. epigenetic therapy

In some patients with sickle cell disease, persistence of expression of fetal hemoglobin into adulthood is associated with reduced severity of the disease. Since butyrate has been shown to increase the production of fetal hemoglobin, it is being studied as a treatment in sickle cell disease.

Question 4

Exon skipping can be induced by
A. Antisense binding to splice sites
B. histone deacetylase inhibitors
C. RNA editing
D. RNA interference
E. Suppressor tRNA

Answer 4

A. Antisense binding to splice sites

Question 5

Chaperone therapy can lead to all of hte following EXCEPT
A. add new protein function
B. allow the target cell to escape apoptosis
C. decrease ERAD
D. restore adequate function of a plasma protein
E. restore adequate secretion of a functional protein

Answer 5

A. add new protein function