Three major approaches exist for targeted CF treatments known as potentiators
, correctors, and production correctors (read-through agents) (68, 69).
Konstan et al., "A CFTR corrector (lumacaftor) and a CFTR potentiator
(ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial," The Lancet Respiratory Medicine, vol.
Hoffmann et al., "Functional defect of variants in the adenosine triphosphate-binding sites of ABCB4 and their rescue by the cystic fibrosis transmembrane conductance regulator potentiator
, ivacaftor (VX-770)," Hepatology, vol.
Vance et al., "A subunit-selective potentiator
of NR2C- and NR2D-containing NMDA receptors," Nature Communications, vol.
The EndoC-[beta]H1 pseudoislets mirror exactly the situation prevailing in primary rat islets , where pyruvate is a weak potentiator
of glucose-induced insulin release and lactate, also a metabolite of glycolytic origin, which completely lacks even a minimal potentiating effect on insulin secretion .
Lahey et al., "Rheumatoid factor as a potentiator
of anti-citrullinated protein antibody-mediated inflammation in rheumatoid arthritis," Arthritis & Rheumatology, vol.
It has created an entirely new therapeutic platform called the Potentiator
which, instead of creating a new antibiotic that bacteria may quickly adapt to, aims to improve the potency and enhance the utility of many classes of existing drugs.
4-Hydroxy Isoleucine: A novel amino acid potentiator
of insulin secretion.
The other major event is the anticipated Food and Drug Administration approval of a fixed-dose combination of ivacaftor and lumacaftor, a combined potentiator
and corrector of the CF transmembrane conductance regulator (CFTR).
BabA-mediated adherence is a potentiator
of the Helicobacter pylori type IV secretion system activity.
Most recently, Lgr4 was shown to be a receptor for Norrin, another canonical Wnt signaling potentiator
, providing another means by which Lgr4 modulates Wnt signaling.