Pharmacotherapy of Cystic Fibrosis: Difference between revisions
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===== Potentiators ===== | ===== Potentiators ===== | ||
Example VX-770 | Example: VX-770 | ||
Attempt to correct Class III and Class IV [[CFTR|CFTR]] mutations by increasing the conductance of the [[CFTR|CFTR]] channel. | Attempt to correct Class III and Class IV [[CFTR|CFTR]] mutations by increasing the conductance of the [[CFTR|CFTR]] channel. | ||
| Line 11: | Line 11: | ||
===== Correctors ===== | ===== Correctors ===== | ||
Example Trimethyl-oxide | Example: Trimethyl-oxide | ||
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 ===== | |||
Example: Gentamicin, G418 | |||
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 13:03, 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, G418
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.