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Life Science 4 > Lecture 23 > Flashcards

Lecture 23 Flashcards

1
Q

Introducing genes to treat diseases

A

gene therapy

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2
Q

Germ-line gene therapy: genes are introduced into the germ cells to – future generations.

A

genetically alter

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3
Q

Somatic cell gene therapy: normal genes are added to the – of at least some of the body’s cells to ameliorate the effects of a defective gene

A

nuclei

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4
Q

Some Goals of Gene Therapy:

Replace – genes with normal genes to cure diseases

A

defective

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5
Q

Some Goals of Gene Therapy: introduce genes which will enhance –

A

immune defenses

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6
Q

Some Goals of Gene Therapy: introduce – into cancer cells

A

suicide genes

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7
Q

Some Goals of Gene Therapy: alter – of genes in the cell

A

regulation

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8
Q

Some Goals of Gene Therapy: stop the replication of –

A

viruses

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9
Q

Genes can be introduced to cells in culture (ex vivo) or –

A

in the patient (in vivo)

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10
Q

+ Adenovirus

A

Infects many cell types

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11
Q
  • Adenovirus
A

Does not integrate into host genome and can be lost.

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12
Q

+ Adeno-associated virus (AAV)

A

Integrates into host genome at specific site and cannot be lost

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13
Q
  • Adeno-associated virus (AAV)
A

difficult to work with

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14
Q
  • Simple retrovirus
A

integrate near genes

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15
Q

+ complex retrovirus (lentivirus)

A

Integrates into host genome and cannot be lost

Integration is not random – occurs away from genes

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16
Q

+ Herpes Simplex Virus (HSV)

A

DNA stays in nucleus without integrating into genome

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17
Q
  • Herpes Simplex Virus (HSV)
A

only infects nervous system cells

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18
Q

Non-viral methods of gene transfer

A

liposome/lipolpex or naked DNA

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19
Q

– are spheres of lipid molecules surrounding an aqueous interior (bilayer); limited amount of DNA will fit in it

A

liposome

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20
Q

– are lipids with positive charge so attract both DNA and cell wall
No immune response, but not as efficient as virus at transferring DNA

A

lipolplex

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21
Q

Direct injection of – into muscle cells was found to be expressed
Could be used to make insulin, blood clotting factors or as vaccines to viruses.

A

naked DNA

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22
Q

Ex vivo usually targets – which are stem cells that generate all other blood cells

A

hematopoietic stem cells (HSC)

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23
Q

X-linked severe combined immunodeficiency (SCID-X1)
β-thalessemia
WAS – an immune difficiency disorder
are associated with

A

HSC

24
Q

is an RNA-mediated mechanism for regulating gene expression in eukaryotes (regularly used to silence specific genes)

A

RNA interference

25
Q

RNAi is a cellular defense against viruses and –

A

transposable elements

26
Q

viral RNA uses – to become dsRNA virus

A

RNA dependent RNA polymerase

27
Q

– cuts dsRNA into 21-25 bp fragments

A

Dicer

28
Q

siRNA fragments created by Dicer bind to – which denatures one of the two strands

A

RISC

29
Q

guide RNA remains bound to RISC and then the – slices other RNA that is complementary to the guide strand

A

argonaute

30
Q

RNAi is used in research to test hypothesis of gene function this is known as –

A

knock-down

31
Q

RNAi is used in therapeutics to – RNA of overexpressed genes

A

degrade

32
Q

RNA is used in therapeutics to target specific –

A

viruses (HIV)

33
Q

T/F: RNAi destroys genome

A

false

34
Q

RNAi methods: – of siRNAs directly into cells

A

transfection

35
Q

RNAi method: introduction of – synthesizes RNA identical to endogenous gene of interest (siRNAs made in organism)

A

vectors

36
Q

T/F: small interfering RNA (siRNA) come from exogenous sources or from other endogenous transcription

A

true

37
Q

Abnormal over-expression of – lead to diseases such as age-related macular degeneration (AMD)

A

VEGF (vascular endothelial growth factor)

38
Q

VEGF is a protein required for blood vessel formation
Excessive blood vessels damage retina
– VEGF should solve the problem

A

silencing

39
Q

problems associated with RNAi include the – of sRNAs and adequate delivery systems

A

stability

40
Q

In RNAi silencing, specificity is provided by –

A

siRNA

41
Q

go in and make changes at a particular site, generally not replacing large amounts

A

genome editing

42
Q

ancient bacterial immune system which identifies the DNA of invading viruses and sends in an endonuclease, like Cas9, to shew it up

A

CRISPRS

43
Q

– can be reprogrammed to recognize nearly any sequence of DNA
just need the right RNA sequence to search a strand of DNA for where to cut

A

Cas9

44
Q

The – regions are homologous to viruses

A

spacer

45
Q

The – are important for binding the Cascade complex

A

repeats

46
Q

Spacer RNA directs complex to – DNA or RNA

A

homologous

47
Q

In E.coli system, cascade complex has – activity

A

Dnase

48
Q

In Pyrococcus furiosus system, Cmr-complex has – activity

A

Rnase

49
Q

One can now use the CRISPR system in eukaryotes to do gene knockouts, gene replacements, and –

A

gene “drive”

50
Q

In crop plants, the genome can be edited and then the – can be segregated away, leaving the plant transgene free

A

Cas9 plasmid

51
Q

selfish genes that appear more frequently in offspring than normal genes, which have about a 50-50 chance of being passed on

A

gene drives

52
Q

T/F: A gene drive always ends up in all offspring, even if only one parent has it;
if given enough generations, it will eventually spread through the entire population

A

true

53
Q

Gene drive: Fight Malaria by changing the mosquitos that are part of their life cycle.
One group modified females to become –

A

sterile

54
Q

Gene drive: Fight Malaria by changing the mosquitos that are part of their life cycle. The other group modified mosquitos by inserting two – directed against the malarial parasite.

A

antibody genes

55
Q

Transgenic to fight Zika virus larvae died due to high expression of –

A

lethal transgene

Life Science 4 (11 decks)

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