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Gene therapy > AAVs > Flashcards

AAVs Flashcards

1
Q

What is the adeno-associated virus genome?

A

ssDNA 4.7kb

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2
Q

What are their promoters?

A

p5 and p19

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3
Q

Where does Rep gene intergrate?

A

chromosome 19

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4
Q

What life cycle do AAVs have?

A

Latent until adeno/herpes infection into the cell.

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5
Q

How do AAVs replicate?

A

Insert into genome via site specific inverted terminal repeats found at either end of genome

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6
Q

What are 4 pros of AAVs?

A

Not pathogenic, no viral gene expression, wide range of cells and intergrate into genome.

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7
Q

What are 3 cons to AAVs?

A

Limited packaging, Rep and helper virus required

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8
Q

AAV needs EA1 gene for what?

A

Activates AAV gene expression

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9
Q

AAV needs E1B and E4 for what?

A

stabilise genome and facilitate transport

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10
Q

AAV gene needs E2A for?1

A

efficient AAV RNA splicing and transcription

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11
Q

AAV gene needs VA RNAs for what?

A

Stimulates efficient transltion

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12
Q

What 5 adeno genes do AAVs need?

A

E1A, E1B, E2A, E4 and VA RNAs.

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13
Q

How main strains of AAV are there?

A

6

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14
Q

What tissues does AAV2 infect?

A

neuronal

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15
Q

What does AAV5 infect?

A

Epidermal

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16
Q

What is immune reaction?

A

Produces many neutralising antibodies cannot readminister

17
Q

What must be in a fake AAV genome?

A

Packaging sequence, terminal repeat, Reb, therapeutic gene.

18
Q

Why does Reb need to be included in AAV vector?

A

Without Red AAV will intergrate randomly at sites of high translation eg chromosome 12

19
Q

How big can therapeutic gene be?

A

2.3kb

20
Q

How can a larger target gene be incorportated?

A

2 AAVs splice them together to make 1 mRNA

21
Q

What are AAV2s good at treating?

A

AAVs 2 containg 7/8 cap gene treating Haemophillia

22
Q

What factors is haemophilia due to?

A

Factors 8 (85%) and 9

23
Q

What can AAVs treat?

A

Haemophillia and retinal degreneration?

Gene therapy (2 decks)

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