Pharmacotherapy of Cystic Fibrosis: Difference between revisions
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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> | 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 | ||
| Line 15: | Line 15: | ||
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<ref>http://www.ptcbio.com/3.1.1_genetic_disorders.aspx</ref>) | Example: Gentamicin, G41, PTC124 (Ataluren<ref>http://www.ptcbio.com/3.1.1_genetic_disorders.aspx</ref>) | ||
| Line 21: | Line 21: | ||
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: | [[Image:Ptc technology white-with-l.gif|535x356px]]<ref>http://www.ptcbio.com/3.1.1_genetic_disorders.aspx</ref> | ||
<br> | <br> | ||
== References<br> == | |||
<references /><br> | <references /><br> | ||
Latest revision as of 14:52, 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.[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