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Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study
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Development of novel therapeutics for all individuals with CF (the future goes on)

Margarida D Amaral1, Patrick T Harrison2

  • 1BioISI - Biosystems & Integrative Sciences Institute, Faculty of Sciences, University of Lisboa, Portugal.

Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society
|November 1, 2022
PubMed

Insights

New treatments for Cystic Fibrosis (CF) show promise, but not all patients benefit. Further research is needed to identify new drug targets and develop gene-editing therapies to reach everyone with CF.

Area of Science:

  • Biomedical research
  • Genetics
  • Pharmacology

Background:

  • Cystic Fibrosis (CF) treatments have advanced, yet significant challenges remain in providing curative therapies to all affected individuals.
  • A critical gap exists in addressing the diverse genetic mutations underlying CF and ensuring equitable access to novel treatments.

Approach:

  • Exploring high-throughput screening methods to uncover mechanistic defects caused by various CF mutations.
  • Investigating innovative gene-editing strategies to correct CFTR gene mutations directly within the patient's genome.

Key Points:

  • Novel therapeutic strategies are essential to overcome limitations in current Cystic Fibrosis treatment paradigms.
  • Identifying new drug targets through holistic, high-throughput approaches is crucial for diverse mutation classes.
  • Gene-correction therapies offer a promising avenue for addressing the root cause of CF at the genomic level.

Conclusions:

  • Despite progress, achieving universal treatment for Cystic Fibrosis requires addressing unmet needs in therapeutic accessibility and efficacy.
  • Continued innovation in drug discovery and gene-based therapies is vital to ensure all individuals with CF can benefit from advanced treatments.
Keywords:
CFTRCystic fibrosisGenome editingTheratypes

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