Pharm #22 - Gene Therapy

Front 1

What are the three types of gene therapy?

Back 1

Replacement, transfer, and augmentation.

Front 2

This type of gene therapy replaces a defective gene with a normal copy of the same gene.

Back 2

Gene replacement therapy.

Front 3

This type of gene therapy transfers a normal copy of a gene into a cell with a defective/abnormal copy of the same gene.

Back 3

Gene transfer therapy.

Front 4

This type of gene therapy uses anti-sense oligonucleotides, si-rna, and sh-rna to inactivate a specific mrna.

Back 4

Gene augmentation therapy.

Front 5

What two types of diseases are potential targets for gene therapy? Give an example of each?

Back 5

Genetic (ex: cystic fibrosis) and acquired (ex: atherosclerosis).

Front 6

How are ex vivo and in vivo gene therapies different?

Back 6

Ex vivo removes the cell with the defective/abnormal gene, transfers the genes, then replaces the cell. In vivo transfers the gene directly into the patient.

Front 7

Why is replacement therapy more difficult than transfer therapy?

Back 7

RT has a insufficient targeted insertion. TT does not require a targeted insertion.

Front 8

What is the disadvantage to gene regulation therapy?

Back 8

It is hard to specifically activate one particular mrna.

Front 9

Name the five viruses commonly used as gene therapy vectors.

Back 9

Retro, lenti, herpes, adeno, and adeno-associated.

Front 10

This potential gene therapy vector is stable and efficient but only infects dividing cells and integrates randomly.

Back 10

Retroviruses.

Front 11

This potential gene therapy vector causes no host immune response and is stable but it is not widely used.

Back 11

Lentivirus.

Front 12

This potential gene therapy vector infects non-dividing cells and has a large genome but is not widely used and has possible expression latency.

Back 12

Herpesvirus.

Front 13

This potential gene therapy vector is easy to prepare and infects non-dividing cells but does not integrate into host genome and can stimulate an immune response.

Back 13

Adenovirus.

Front 14

This potential gene therapy vector is stable and non-pathogenic but is difficult to prepare with a small genome.

Back 14

Adeno-associated virus.

Front 15

What are the advantages and disadvantages of using non-viral vectors for gene therapy?

Back 15

A - easy to prepare, not immunogenic, and there is no limit on gene size. D - lack of targeting specificity.

Front 16

Name the seven challenges facing gene therapy.

Back 16

Ethical issues (it should be therapy), risk of cancer, short-lived (may need multiple treatments), unwanted immune response, viral vectors, death, and negative perception.