Pharmacotherapy of Cystic Fibrosis
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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>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 | Example: VX-770 | ||
| Line 9: | Line 9: | ||
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. | ||
| − | + | == Correctors == | |
Example: Trimethyl-oxide | Example: Trimethyl-oxide | ||
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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 == | |
| − | 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. |
| + | |||
| + | [[Image:Ptc technology white-with-l.gif|535x356px]]<ref>http://www.ptcbio.com/3.1.1_genetic_disorders.aspx</ref> | ||
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| + | <br> | ||
| + | |||
| + | == References<br> == | ||
| + | |||
| + | <references /><br> | ||
Latest revision as of 14:52, 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.[1]
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[2])
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.
References
- ↑ 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
- ↑ http://www.ptcbio.com/3.1.1_genetic_disorders.aspx
- ↑ http://www.ptcbio.com/3.1.1_genetic_disorders.aspx
