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Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study
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Proteostasis Regulators in Cystic Fibrosis: Current Development and Future Perspectives

Irene Brusa1,2, Elvira Sondo3, Federico Falchi4

  • 1Department of Pharmacy and Biotechnology, University of Bologna, 40126 Bologna, Italy.

Journal of Medicinal Chemistry
|April 4, 2022
PubMed

Insights

New drugs targeting cystic fibrosis (CF) aim to improve CF transmembrane conductance regulator (CFTR) protein function. Researchers are exploring proteostasis regulators to enhance current CFTR corrector and potentiator therapies.

Area of Science:

  • Molecular Biology
  • Pharmacology
  • Genetics

Background:

  • Cystic fibrosis (CF) is characterized by the F508del mutation in the CF transmembrane conductance regulator (CFTR) protein.
  • This mutation causes protein misfolding and premature degradation, impairing CFTR function.
  • Current CFTR potentiators and correctors offer limited clinical benefit, necessitating novel therapeutic strategies.

Purpose of the Study:

  • To review recent advancements in the discovery of CFTR proteostasis regulators.
  • To discuss the design, chemical structure, and structure-activity relationships of these novel agents.
  • To explore the potential of modulating the CFTR proteostasis environment for enhanced CF therapy.

Main Methods:

  • Literature review of recent research on CFTR proteostasis regulators.
  • Analysis of drug design, chemical structures, and structure-activity relationships.
  • Discussion of therapeutic opportunities and challenges in the field.

Main Results:

  • Several novel CFTR proteostasis regulators have been identified.
  • These agents show promise in enhancing the efficacy of existing CFTR-targeting drugs.
  • Understanding structure-activity relationships is crucial for optimizing therapeutic potential.

Conclusions:

  • Modulating CFTR proteostasis represents a promising avenue for improving cystic fibrosis treatment.
  • Further research into CFTR proteostasis regulators is warranted to overcome current therapeutic limitations.
  • This field offers significant opportunities for developing more effective CF therapies.

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