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pa30287 > Biologicals 3 > Flashcards

Biologicals 3 Flashcards

1
Q

What was Macugen?

A

The first aptamer drug and first RNA drug

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2
Q

What was Macugen used for?

A

Treatment of ‘wet’ age related macular degeneration

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3
Q

How does Macugen work?

A

Binds to VEGF, reducing blood vessel growth.

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4
Q

How is Macugen given?

A

IV injection every 6 weeks

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5
Q

What can RNAi do?

A

Knock down gene expression if sequence is known

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6
Q

What is miRNA derived from?

A

Genes that specifically code miRNAs

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7
Q

What can happen to cells with missing miRNAs?

A

DNA coding missing miRNAs could be delivered to cells to replace the missing miRNAs

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8
Q

Overexpressed miRNAs can be inhibited by what?

A

anti-miRNAs, complementary nucleotides that bind to the problem miRNA

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9
Q

What are vital in the boody’s defence against tumours?

A

Cytotoxic T lymphocytes

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10
Q

What to CTLs recognise?

A

Short antigenic peptides in association with major histocompatibility complex molecules displayed on the cell surface

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11
Q

Bone marrow derived dendritic cells display what to immature CTLs to activate them?

A

MHC-peptide complexes

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12
Q

What are the 4 main genetic therapy targets?

A

Cancer
Cardiovascular disease
Monogenic disease
Infectious diseases

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13
Q

What are the challenges to delivery of genetic material?

A
  1. Complex formation
  2. Transport from delivery site
  3. Cellular uptake
  4. Endosome release
  5. Trafficking
  6. Nuclear entry
  7. Transcription
  8. mRNA export
  9. Translocation
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14
Q

What can retroviruses be used to target?

A

Specific cells

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15
Q

What is the risk of using retroviruses?

A

Tumour development

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16
Q

Are adenoviruses ds or ssDNA?

A

dsDNA

17
Q

Are retroviruses ssRNA or dsDNA?

A

ssRNA

18
Q

Adenovirus infects divinding or non-dividing cells?

A

Both

19
Q

What was Glybera approved for?

A

Treatment of lipoprotein lipase deficiency

20
Q

What are the advantages of viral vectors?

A

High transfer efficiency

Some inherent tissue tropisms

21
Q

What are the disadvantages of viral vectors?

A

Limited DNA/RNA capacity.

Difficult production.

22
Q

What physical techniques can be used to create non-viral nucleic acid delivery?

A

Direct injection
Gene gun
Ultrasound
Electroporation

23
Q

What chemical techniques can be used to create non-viral nucleic acid delivery?

A

Encapsulation

Complexation

24
Q

What are the advanatges of non-viral vectors?

A

Easy to prepare and modify.
Reduced immunogenicity.
Large capacity

25
Q

What are the disadvantages of non-viral vectors?

A

Poor efficiency

Transient

26
Q

What are the advantages of lipoplexes in vitro?

A

Commercialised DNA transfection reagents are avialable

27
Q

What are the disadvantages to lipoplexes in vivo?

A

High positive charge, toxic, short circulation, large particle size, unstable

pa30287 (17 decks)

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