Ch 5

Question 1

gene therapy

Answer 1

using DNA as a therapeutic (in contrast to drug therapy)

Question 2

in general, a mutated gene must be fixed by either…

Answer 2

a) adding the normal gene or b) replacing the nutated gene with a normal gene

Question 3

how are cancer cells killed?

Answer 3

using genes

Question 4

what are DNA vaccines?

Answer 4

genes to combat infection disease

Question 5

somatic cells

Answer 5

cells derived from the basic germ layers of the body that DO NOT pass their genetic information to the next generation

Question 6

germ cells

Answer 6

cells that give rise to the gametes and DO pass their genetic information to the next generation

Question 7

gastrulation

Answer 7

forms the basic “germ layers” of the body and creates the germ cells for the next generation

Question 8

direct delivery of somatic cell gene therapy

Answer 8

packaged into a delivery vehicle such as a retrovirus and injected into the patient

Question 9

somatic cell gene therapy

Answer 9

a) genetically identical iPSCs are generated from the patient B) adult stem cells are isolated and propagated in the laboratory

Question 10

cell based delivery

Answer 10

therapeutic gene is packaged into a delivery vehicle such as a retrovirus and introduced into the cells

Question 11

challenges of somatic gene therapy

Answer 11

must get therapeutic gene into relevant cells’ nucleus, gene must be expressed long term, safety concerns

Question 12

in direct delivery, a “delivery vehicle” such as what is used to target the gene to the tissue

Answer 12

inactive virus

Question 13

foreign genes tend to be what?

Answer 13

“silenced”

Question 14

safety concerns to somatic gene therpay

Answer 14

removal of engineered cells could prove difficult, 1999 death due to a massive adverse immune response to virus, side affects from the integration of foreign DNA

Question 15

what is the U.S. policy on somatic cell gene therapy?

Answer 15

FDA has approved experimental trials, but no products have been approved

Question 16

in 2000, a sever combined immune disease (SCID) gene therapy trial resulted in what?

Answer 16

development of leukemia in some of the treated patients

Question 17

research in somatic cell gene therapy

Answer 17

development of gene delivery vehicles that don’t rely on viruses, new ways to destroy, introduce, or repair genes, demonstrating treatments in animal models, using patient-specific induced pluripotent stem cells (iPSCs) to correct genetic defects and then creating the appropriate tissue

Question 18

reproductive (germline) gene therappy

Answer 18

gene therapy to alter the genetic constitution of germ cells

Question 19

what does gene therapy do?

Answer 19

affects all cells of the body, not targeted to a specific tissue, manipulates the genome of a person before they are born, imposes a permanent change that is passed on to subsequent generations

Question 20

germline gene therapy: making “transgenic animals”

Answer 20

1) deliver a gene into the nucleus of a fertilized one cell embryo (zygote) 2) develop to blastocyst and implant into a female uterus 3) test to verify the gene is present in the offspring

Question 21

animals can be engineered to produce 2-3 times more what?

Answer 21

muscle mass

Question 22

transgenics work well for what?

Answer 22

adding a gene

Question 23

to repair or replace a gene with transgenics currently requires what?

Answer 23

cells that can be cultured in a dish

Question 24

germline gene therapy via reproductive cloning:

Answer 24

1) genetically modify skin cells, 2) perform SCNT, 3) implant

Question 25

germline gene therapy via “parentless children”

Answer 25

1) genetically modify iPSCs 2) make sperm and egg from the iPSCs 3) fertilize and implant

Question 26

moral and safety bar for germline engineering

Answer 26

changing genes that make up the “human genome”, changing course of human evolution, designer babies

Question 27

chimera

Answer 27

single organism composed of genetically distinct cells that originated from different zygotes

Question 28

human chimera requires what?

Answer 28

multiple fertilized embryos - IVF implantation of multiple embryos will increase the frequency of human chimerism

Question 29

how were chimeric monkeys generated?

Answer 29

by mixing cleavage-stage embryonic cells from several different monkey embryos

Question 30

what is the goal of the use of human-animal chimeras in biomedical research?

Answer 30

create animals that carry human organs for research and transplantation

Question 31

in general, inter-species chimeras will work with what?

Answer 31

closely related species

Question 32

has there been a foundation set for chimera research regulation in the US?

Answer 32

no