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.Cite error: Invalid <ref> tag; name cannot be a simple integer. Use a descriptive title

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[1])

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.


References

  1. http://www.ptcbio.com/3.1.1_genetic_disorders.aspx

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