View source for 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.<ref>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  ==

Example: VX-770 

Attempt to correct Class III and Class IV [[CFTR|CFTR]] mutations by increasing the conductance of the [[CFTR|CFTR]] channel. 

== Correctors  ==

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]]. 

== Termination Suppressors  ==

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. 

[[Image:Ptc technology white-with-l.gif|535x356px]]<ref>http://www.ptcbio.com/3.1.1_genetic_disorders.aspx</ref> 

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== References<br>  ==

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