Pharmacotherapy of Cystic Fibrosis: Difference between revisions
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Attempt to correct Class II [[CFTR|CFTR]] mutations by improving the trafficking of [[CFTR|CFTR]] channels to the [[Lipid bi-layer|Cell Membrane]]. | Attempt to correct Class II [[CFTR|CFTR]] mutations by improving the trafficking of [[CFTR|CFTR]] channels to the [[Lipid bi-layer|Cell Membrane]]. | ||
===== Termination Suppressors ===== | ===== Termination Suppressors ===== | ||
Example: Gentamicin, | Example: Gentamicin, G41, PTC124 (Ataluren) | ||
Suppress [[ | Suppress [[Premature Stop Codon|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. | ||
Revision as of 13:04, 13 November 2010
Pharmacotherapy of Cystic Fibrosis
Pharmacotherapy relates to the use of chemical drugs to regulate and improve the function of defective CFTR channels.
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)
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.