Pharmacotherapy of Cystic Fibrosis

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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  =====
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== Potentiators  ==
  
 
Example: VX-770  
 
Example: VX-770  
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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  =====
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== 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  =====
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== 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>)  
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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]]
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[[Image:Ptc technology white-with-l.gif|535x356px]]<ref>http://www.ptcbio.com/3.1.1_genetic_disorders.aspx</ref>
  
<br>
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<br>  
  
=== References<br>  ===
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== References<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.

Ptc technology white-with-l.gif[3]


References

  1. 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
  2. http://www.ptcbio.com/3.1.1_genetic_disorders.aspx
  3. http://www.ptcbio.com/3.1.1_genetic_disorders.aspx

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