Pharmacotherapy of Cystic Fibrosis

From The School of Biomedical Sciences Wiki
(Difference between revisions)
Jump to: navigation, search
 
(7 intermediate revisions by one user not shown)
Line 1: Line 1:
 
== 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  =====
+
== 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  =====
+
== 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 =====
+
== Termination Suppressors ==
  
Example: Gentamicin, G418
+
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>
 +
 
 +
<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

Personal tools
Namespaces
Variants
Actions
Navigation
Toolbox