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>  
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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 name="2">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  =====
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===== Termination Suppressors  =====
 
===== Termination Suppressors  =====
  
Example: Gentamicin, G41, PTC124 (Ataluren)  
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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.
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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.  
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<br>
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=== References<br>  ===
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<references /><br>

Revision as of 13:26, 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.Cite error: Invalid <ref> tag; name cannot be a simple integer. Use a descriptive title

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[1])

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

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

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