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Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients
07:04

Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients

Published on: February 11, 2017

Small molecules to treat cystic fibrosis

Michael Wilschanski1

  • 1Pediatric Gastroenterology Unit, Division of Pediatrics, Hadassah Hospitals, Hebrew University, Jerusalem, Israel 91240. michaelwil@hadassah.org.il

Proceedings of the American Thoracic Society
|October 30, 2010
PubMed

Insights

Mutation-specific therapies for cystic fibrosis (CF) are advancing. New treatments target the basic gene defect, focusing on potentiators, correctors, and premature termination codon suppressors, with promising preclinical and clinical trial results.

Area of Science:

  • Genetics and Molecular Biology
  • Pulmonology
  • Pharmacology

Background:

  • Cystic Fibrosis (CF) is a genetic disorder caused by mutations in the CFTR gene.
  • Over 21 years since gene discovery, understanding of CF pathophysiology has significantly improved.
  • This enhanced knowledge facilitates the development of targeted therapeutic strategies.

Purpose of the Study:

  • To summarize current knowledge on mutation-specific therapies for cystic fibrosis.
  • To focus on orally bioavailable potentiators and correctors.
  • To review suppressors of premature termination codons in CF.

Main Methods:

  • Review of preclinical model systems for CF.
  • Analysis of ongoing and completed clinical trials for CF therapies.
  • Focus on therapies targeting the underlying genetic defect in CF.

Main Results:

  • Significant progress in understanding CF pathophysiology driven by gene mutations.
  • Development of novel therapeutic approaches targeting the basic defect of CF.
  • Emerging orally bioavailable potentiators and correctors show promise.

Conclusions:

  • Mutation-specific therapies represent a new frontier in CF treatment.
  • Potentiators, correctors, and PTC suppressors are key areas of focus.
  • Preclinical and clinical data support the potential of these advanced CF treatments.

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