Pharmacotherapy of Cystic Fibrosis
From The School of Biomedical Sciences Wiki
(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. | ||
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===== 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
Contents |
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.
