11 - drug discovery 3 Flashcards

1
Q

What is the MOA of antisense oligonucleotides (ASO)?

A

Blocks the mRNA directly to reduce amount of protein made

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2
Q

Example of an ASO.

A

Patisiran (Onpattro)

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3
Q

What is Patisiran (Onpattro)?

A

RNAi for hereditary transthyretin-mediated amyloidosis

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4
Q

What is the MOA of mRNA medicine?

A

Introduces exogenous mRNA into body, so cells can make proteins based on introduced mRNA

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5
Q

Example of mRNA medicine.

A

COVID-19 vaccine

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6
Q

What is required in a mRNA medicine?

A
  • mRNA sequence
  • “Right” coating
  • RNA sequence
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7
Q

What is the MOA of monoclonal antibody and antibody-drug conjugate?

A
  • A type of protein that specifically binds to its target to block the function of that target protein
  • Or help recognize a specific group of cells that express that target protein
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8
Q

Example of monoclonal antibody.

A

PD1 and PD-L1

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9
Q

How does gene therapy with adeno-associated virus (AAV) work?

A
  • Transgene is packaged into AAV vectors
  • Through an infusion, AAVs carrying the transgene targets the liver
  • AAV vectors deliver transgene to nucleus of liver cells to enable production of therapeutic protein
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10
Q

What is an example of a drug that is a gene therapy w adeno-associated virus?

A

Zolgensma

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11
Q

What is Zolgensma?

A

A biologic drug consisting of adeno-associated virus (AAV9) capsids that contain SMN1 transgene along w synthetic promoters to tx spinal muscular atrophy (SMA)

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12
Q

What is SMA?

A

A neuromuscular disorder caused by a mutation in SMN1 gene, which leads to decrease in SMN protein

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13
Q

What are SMN proteins?

A

A protein necessary for survival of motor neurons

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14
Q

What is CRISPR/Cas9?

A
  • A DNA editing technique
  • Works like a biological version of word-processing program’s “find and replace” function
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15
Q

What does CRISPR stand for?

A

Cluster regularly interspaced short palindromic repeats

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16
Q

How does CRISPR/Cas9 work?

A
  • A cell is transfected w an enzyme containing the following: guide molecule, health DNA copy, DNA cutting enzyme
  • Specifically designed synthetic guide finds target DNA strand
  • Enzyme cuts off target DNA strand
  • Defective DNA strand is replaced w healthy copy
17
Q

How does stem cell therapy work?

A

Has potential to generate any cell types in body to replace damaged tissue

18
Q

What is the current most promising stem cell therapy?

A

Induced pluripotent stem cells (iPSCs)

19
Q

What are other possible stem cell therapies?

A
  • Embryonic stem cells (ESC)
  • Neural stem cells (NSC)
  • Progenitor cells