Translational research to enable personalized treatment of cystic fibrosis

Marne C. Hagemeijer, Daniel J. Siegwart, Lisa J. Strug, Liudmila Cebotaru, Michael J. Torres, Aderonke Sofoluwe, Jeffrey M. Beekman

Research output: Contribution to journalArticle

Abstract

Translational research efforts in cystic fibrosis (CF) aim to develop therapies for all subjects with CF. To reach this goal new therapies need to be developed that target multiple aspects of the disease. To enable individuals to benefit maximally from these treatments will require improved methods to tailor these therapies specifically to individuals who suffer from CF. This report highlights current examples of translational CF research efforts to reach this goal. The use of intestinal organoids and genetics to better understand individual assessment of CFTR modulator treatment effects to ultimately enable a better personalized treatment for CF subjects will be discussed. In addition, development of viral vectors and non-viral synthetic nanoparticles for delivery of mRNA, sgRNA and DNA will be highlighted. New approaches to restore function of CFTR with early premature termination codons using nanoparticle delivery of suppressor tRNAs and new insights into mechanisms of airway epithelial repair will be reviewed as well. The state-of-the-art approaches that are discussed in this review demonstrate significant progress towards the development of optimal individual therapies for CF patients, but also reveal that remaining challenges still lie ahead.

Original languageEnglish (US)
JournalJournal of Cystic Fibrosis
DOIs
StateAccepted/In press - Jan 1 2017

Fingerprint

Translational Medical Research
Cystic Fibrosis
Nanoparticles
Therapeutics
Organoids
Nonsense Codon
Transfer RNA
Messenger RNA
DNA
Research

Keywords

  • CFTR modulator
  • Cystic fibrosis
  • Gene therapy
  • Nanoparticles
  • Personalized medicine
  • Translational research
  • TRNA

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Pulmonary and Respiratory Medicine

Cite this

Hagemeijer, M. C., Siegwart, D. J., Strug, L. J., Cebotaru, L., Torres, M. J., Sofoluwe, A., & Beekman, J. M. (Accepted/In press). Translational research to enable personalized treatment of cystic fibrosis. Journal of Cystic Fibrosis. https://doi.org/10.1016/j.jcf.2017.10.017

Translational research to enable personalized treatment of cystic fibrosis. / Hagemeijer, Marne C.; Siegwart, Daniel J.; Strug, Lisa J.; Cebotaru, Liudmila; Torres, Michael J.; Sofoluwe, Aderonke; Beekman, Jeffrey M.

In: Journal of Cystic Fibrosis, 01.01.2017.

Research output: Contribution to journalArticle

Hagemeijer, Marne C. ; Siegwart, Daniel J. ; Strug, Lisa J. ; Cebotaru, Liudmila ; Torres, Michael J. ; Sofoluwe, Aderonke ; Beekman, Jeffrey M. / Translational research to enable personalized treatment of cystic fibrosis. In: Journal of Cystic Fibrosis. 2017.
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