cystic fibrosis 12Therapeutic strategies for CF

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1. mucolytics: select incorrect mucolytic

  • High frequency oscillation; to disrupt physical entanglements (types 4+5)
  • Deoxyribonuclease; (DNase, Pulmozyme, Dornase alfa), depolymerises DNA
  • elexacaftor
  • Sulphydryl reagents; target -S-S- bonds between mucin molecules, eg dithiothreitol, N-acetyl cysteine (type 1)
  • Heparin, dextran sulphate; highly ionic, negatively charged, molecules that disrupt ionic and hydrogen bonds (types 2+3)
  • Mannitol; hyperosmotic to rehydrate the airways
  • Hypertonic saline
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2. to modify the basic defect of CF, you can alter other channels e.g Na, name a drug that can do this? (but not majorly effective solo)

  • CFTR modulators
  • elexacaftor
  • amiloride
  • mannitol

3. what can activate mutant CFTR?

  • mannitol
  • Gene therapy
  • CFTR modulators
  • elexacaftor

4. many CFTR modulating drugs, varying from potentiators, to correctors to combos of the 2: select incorrect answer

  • Orkambi
  • elexacaftor
  • Kaftrio
  • Symkevi
  • tezacaftor
  • or (Kalydeco-2016)
  • lumacaftor
  • mannitol
  • tezacaftor

5. NSAIDs also not effective as they induce the synthesis of pro-inflammatory cytokines. Anti-protease. Neutrophil elastase is an important target for therapy. - Therapies based on the natural elastase inhibitors, include recombinant alpha1-antitrypsin (alp

  • neutrophil elastase should outweigh antiproteases
  • antiproteases should outweigh neutrophil elastase
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