Pharmacotherapy of Cystic Fibrosis: Difference between revisions
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== Pharmacotherapy of Cystic Fibrosis == | == Pharmacotherapy of Cystic Fibrosis == | ||
Pharmacotherapy relates to the use of chemical drugs to regulate and improve the function of defective [[Cystic fibrosis|CFTR]] channels.<br> | Pharmacotherapy relates to the use of chemical drugs to regulate and improve the function of defective [[Cystic fibrosis|CFTR]] channels.<ref name="2">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.</ref><br> | ||
===== Potentiators ===== | ===== Potentiators ===== | ||
| Line 17: | Line 17: | ||
===== Termination Suppressors ===== | ===== Termination Suppressors ===== | ||
Example: Gentamicin, G41, PTC124 (Ataluren) | Example: Gentamicin, G41, PTC124 (Ataluren<ref>http://www.ptcbio.com/3.1.1_genetic_disorders.aspx</ref>) | ||
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. | 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. | ||
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=== References<br> === | |||
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Revision as of 13:26, 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.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.