Module 12

Question 1

enzyme replacement therapy

Answer 1

recombinant enzyme infusion

Question 2

substrate reduction therapy

Answer 2

oral drug reduces substrate so enzyme can function more effectively

Question 3

pharmacological chaperone therapy

Answer 3

oral drug binds to misfolded protein, restoring function

Question 4

gene therapy

Answer 4

therapy uses genes directly or targets genes

Question 5

direct delivery

Answer 5

therapeutic gene packaged into delivery vehicle and injected

Question 6

cell-based delivery

Answer 6

therapeutic gene packaged into a vehicle, introduced to in vitro stem cell, reintroduced to patient

Question 7

germline gene therapy

Answer 7

alters DNA of fertilized egg for all cells to be affected. not done in humans

Question 8

somatic gene therapy

Answer 8

only somatic cells, not passed down to generations

Question 9

transgenic organisms

Answer 9

combination of DNA from different organisms

Question 10

in vivo

Answer 10

delivered directly to patient

Question 11

ex vivo

Answer 11

delivered to cell then to patient

Question 12

gene therapy vectors

Answer 12

retrovirus, adenovirus, naked DNA, liposome plasmid vector

Question 13

tropism

Answer 13

affinity for cell or tissue type

Question 14

severe combined immune deficiency

Answer 14

deficiency of enzyme ADA leading to toxic buildup of deoxy ATP destroying T/B cells

Question 15

notorious setbacks

Answer 15

19yo with enzyme deficiency toxic buildup of ammonia died after gene therapy; 4 French kids developed leukemia

Question 16

clinicaltrials.gov

Answer 16

list of all gene therapy trials ongoing

Question 17

gene replacement/augmentation therapy

Answer 17

adds functional copy of a gene

Question 18

end-run therapy

Answer 18

target is downstream of a gene that supplies a needed function

Question 19

suppression therapy

Answer 19

targets GOF mutations to stop a bad gene. gene silencing RNAi

Question 20

gene supplementation therapy

Answer 20

increases gene dosage

Question 21

cancer therapy

Answer 21

targets tumor cells while leaving healthy cells

Question 22

CAR T cell therapy

Answer 22

engineered t/b-cell combination, super t-cell

Question 23

gene-based therapy

Answer 23

using genes to produce therapeutic agent

Question 24

ZFN

Answer 24

zinc finger nuclease cuts one gene at a time

Question 25

TALEN

Answer 25

transcription-activator like effector nuclease cuts one gene at a time

Question 26

CRISPR

Answer 26

clustered regularly interspaced short palindromic repeat uses guide RNA to target DNA segment

Question 27

operation warp speed

Answer 27

effort to accelerate vaccine production

Question 28

vaccine

Answer 28

weakened fragment of organisms containing antigen

Question 29

B-cells

Answer 29

create neutralizing antibodies

Question 30

cytotoxic t-cells

Answer 30

recognize infected cells to kill

Question 31

DNA/RNA vaccines

Answer 31

uses DNA/RNA to teach immune system to target viral protiens

Question 32

live attenuated

Answer 32

weakened version of actual virus

Question 33

inactivated

Answer 33

whole virus has been killed

Question 34

subunit

Answer 34

piece of virus surface to focus immune response

Question 35

viral vector

Answer 35

harmless virus to deliver viral genes for immunity

Question 36

poly A tail

Answer 36

influences protein expression, prevents decapping, masks translation

Question 37

perfusion closed conformation

Answer 37

(SARS-CoV-2) triggers immune system