Pharmacotherapy of Cystic Fibrosis

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Pharmacotherapy of Cystic Fibrosis

Pharmacotherapy relates to the use of chemical drugs to regulate and improve the function of defective CFTR channels.[1]

Potentiators

Example: VX-770

Attempt to correct Class III and Class IV CFTR mutations by increasing the conductance of the CFTR channel.

Correctors

Example: Trimethyl-oxide

Attempt to correct Class II CFTR mutations by improving the trafficking of CFTR channels to the Cell Membrane.

Termination Suppressors

Example: Gentamicin, G41, PTC124 (Ataluren[2])

Suppress termination mutations which would normal result in premature termination and truncation of the protein. These drugs are designed to restore 'read-through' and enable the production of normal CFTR.

Ptc technology white-with-l.gif[3]


References

  1. David L. Rimoin, J. Michael Connor, Reed E. Pyeritz, Bruce R. Korf (2007). Emery and Rimoin's Principles and Practice of Medical Genetics e-dition. 5th ed. Amsterdam: Elsevier. p1354-1394
  2. http://www.ptcbio.com/3.1.1_genetic_disorders.aspx
  3. http://www.ptcbio.com/3.1.1_genetic_disorders.aspx

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