RNA editing applied to cystic fibrosis: RESTORE can target G542X CFTR mRNA and revert the nonsense mutation
The obtained results suggest that the RESTORE approach might be explored as a promising strategy to treating nonsense mutations in CFTR, potentially contributing to novel therapeutic options for CF patients.
Authors
- Raffaella Melfi (ORCID: https://orcid.org/0000-0003-4463-8156)
- Aldo Di Leonardo (ORCID: https://orcid.org/0000-0003-4760-7179)
- Simona Titoli
- Silvia Gargano
- Viviana Barra (ORCID: https://orcid.org/0000-0002-7186-4485)
Institutions
- University of Palermo (IT)
Publication Details
- Journal
- Gene
- Published
- 2025-03-05
- DOI
- https://doi.org/10.1016/j.gene.2025.149384
- Citations
- 3
- Primary Topic
- RNA regulation and disease
- Type
- article
- Field-Weighted Citation Impact
- 2.42
Funders
- Università degli Studi di Palermo
- Fondazione per la Ricerca sulla Fibrosi Cistica