Gene, RNA, and ASO-based therapeutic approaches in Cystic Fibrosis

Normand E. Allaire*, Uta Griesenbach, Batsheva Kerem, John D. Lueck, Noemie Stanleigh, Yifat S. Oren

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

3 Scopus citations

Abstract

Most people with Cystic Fibrosis (PwCF) harbor Cystic Fibrosis Transmembrane Conductance (CFTR) mutations that respond to highly effective CFTR modulators (HEM); however, a small fraction of non-responsive variants will require alternative approaches for treatment. Furthermore, the long-term goal to develop a cure for CF will require novel therapeutic strategies. Nucleic acid-based approaches offer the potential to address all CF-causing mutations and possibly a cure for all PwCF. In this minireview, we discuss current knowledge, recent progress, and critical questions surrounding the topic of Gene-, RNA-, and ASO-based therapies for the treatment of Cystic Fibrosis (CF).

Original languageAmerican English
Pages (from-to)S39-S44
JournalJournal of Cystic Fibrosis
Volume22
DOIs
StatePublished - Mar 2023

Bibliographical note

Publisher Copyright:
© 2023 European Cystic Fibrosis Society

Keywords

  • ASO
  • Cystic Fibrosis
  • Delivery
  • Gene
  • RNA
  • Therapeutic

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