Potentiators, Correctors and Production correctors2 of 3
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CFTR modulators are small molecules that target specific defects caused by mutations in the CFTR gene. They are classified into three main groups: Potentiators, Correctors and Production correctors (or Read-through agents). This nomenclature describes their mechanism of action, which is determined by the type of mutation they target.1
Production correctors instruct ribosomes to read-through premature termination codons (PTCs) during mRNA translation.2 This ensures a full-length CFTR protein is produced and is transported to the Golgi for further processing. Remember – PTCs instruct ribosomes to stop translating CFTR protein before it reaches its full length.
Click here to see an animation of this process.
Correctors overcome defective protein processing that normally results in the production of misfolded CFTR. This allows increased trafficking of CFTR to the plasma membrane.3
Potentiators increase the activity of defective CFTR at the cell surface. Potentiators can either act on gating defects or conductance defects.3,4