Sarge 4-28 Gene therapy

Question 1

direct method of gene therapy

Answer 1

gene delivered to cells via virus vehicle

Question 2

cell-based method of gene delivery

Answer 2

stem cells are modified with the gene via virus vehicle, delivered back into the patient

Question 3

3 diseases in which gene therapy is being pursued

Answer 3

CF, diabetes, anemia, SCID, Choroideremia, retinitis pigmentosum

Question 4

3 vectors for gene therapy

Answer 4

liposomes, retroviruses, oncolytic viruses (T-vec)

Question 5

what tissue is targeted when proteins must be present in the bloodstream

Answer 5

liver

Question 6

what is “suicide gene therapy”

Answer 6

when the virus targets a certain cell type, so that when a pro-drug is given to the person, only the cells taking up that virus convert the drug to its toxic form and DIE

Question 7

In what disease would the gene need to be given to multiple tissues?

Answer 7

CF

Question 8

HIV/ Retrovirus vectors

Answer 8

target non-dividing cells, inserts into DNA, disease-associated

Question 9

Herpes Simplex virus

Answer 9

does NOT insert into genome- not infective of whole body, not inserted in DNA (only episomal) disease associated

Question 10

Adenovirus

Answer 10

infects non-dividing cells, does not insert DNA (episomal only), disease-associated

Question 11

Adeno-associated virus

Answer 11

infects non-dividing cells, does not insert into DNA (episomal only), NOT disease-associated

Question 12

Oncolytic virus

Answer 12

virus infects all cells but only replicates and destroys cells expressing biochemical pathways of cancer

Question 13

T-vec mechanism

Answer 13

same as oncolytic viruses, but the dying cancer cells are expressing Granulocyte Macrophage Colony Stimulating Factor (GM-CSF) that attracts dendritic cells to start presenting cancer antigens that attract T-cells