Delivery of ENaC siRNA to epithelial cells mediated by a targeted nanocomplex: a therapeutic strategy for cystic fibrosis
Open accessAbstract
The inhibition of ENaC may have therapeutic potential in CF airways by reducing sodium hyperabsorption, restoring lung epithelial surface fluid levels, airway hydration and mucociliary function. The challenge has been to deliver siRNA to the lung with sufficient efficacy for a sustained therapeutic effect. We have developed a self-assembling nanocomplex formulation for siRNA delivery to the airways that consists of a liposome (DOTMA/DOPE; L), an epithelial targeting peptide (P) and siRNA (R). LPR formulations were assessed for their ability to silence expression of the transcript of the gene encoding the α-subunit of the sodium channel ENaC in cell lines and primary epithelial cells, in submerged cultures or grown in air-liquid interface conditions. LPRs, containing 50 nM or 100 nM siRNA, showed high levels of silencing, particularly in primary airway epithelial cells. When nebulised these nanocomplexes still retained their biophysical properties and transfection efficiencies. The silencing ability was determined at protein level by confocal microscopy and western blotting. In vivo data demonstrated that these nanoparticles had the ability to silence expression of the α-ENaC subunit gene. In conclusion, these findings show that LPRs can modulate the activity of ENaC and this approach might be promising as co-adjuvant therapy for cystic fibrosis.
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LPR nanocomplexes facilitated translocation of siRNA through mucus, mediated efficient ENaCsiRNA transfections in primary CF epithelial cells, and reduced 30% of αENaC and βENaC mRNA.
“Results showed that the established LPR nanocomplexes facilitated the translocation of siRNA through mucus, mediated efficient ENaCsiRNA transfections in primary CF epithelial cells, and reduced 30% of the αENaC and βENaC mRNA.”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [95]
Manunta et al. reported an ENaCsiRNA nanocomplex that induced effective in vitro and in vivo silencing of airway ENaC.
“In 2017, Manunta et al. reported the development of an ENaCsiRNA nanocomplex that induced effective in vitro and in vivo silencing of airway ENaC.[95, 96]”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [95]
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Elboraie, S., Kafetzis, K. N., Shrivastava, R., & Tagalakis, A. D. (2021) · IntechOpen eBooks · doi:10.5772/intechopen.92434 · Cited by 2 · see citing works
Gene delivery using chemical methods
Bhatt, P., Surti, N. I., & Mahajan, A. N. (2025) · Elsevier eBooks · doi:10.1016/b978-0-443-27416-9.00008-3 · Cited by 1 · see citing works
In Utero Gene Editing for Inherited Lung Diseases
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Cystic fibrosis: current treatment and future direction
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- Authors
- Manunta, M. D. I., Tagalakis, A. D., Attwood, M., Aldossary, A. M., Barnes, J. L., Munye, M. M., Weng, A., McAnulty, R. J., & Hart, S. L.
- Affiliations
- University College London (GB)
- University of Oxford (GB)
- Corresponding author
- Hart, Stephen L.
- ORCID
- Venue
- Scientific Reports
- Published
- 2017-04-06
- Volume
- 7
- Issue
- 1
- Pages
- 700-700
- ISSN
- 2045-2322 (ISSN-L)
- PMC
- PMC5428798
- OpenAlex
- W2600042835
- Cited by
- 58
- References
- 38
- Citations by year
- 2026: 2 · 2025: 2 · 2024: 5 · 2023: 2 · 2022: 10 · 2021: 10 · 2020: 10 · 2019: 8 · 2018: 6 · 2017: 3
- FWCI
- 3.00
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Locations
- Scientific Reports (publishedVersion, cc-by)
- PubMed Central (publishedVersion, cc-by)
- PubMed (publishedVersion)
- Edge Hill University Research Information Repository (Edge Hill University) (publishedVersion, cc-by)
- Edge Hill University (submittedVersion)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
- UCL Discovery (University College London) (submittedVersion)
- Refubium (Universitätsbibliothek der Freien Universität Berlin) (submittedVersion, cc-by)
- Funders
- Wellcome Trust (WT094348MA)
- NIHR Biomedical Research Centre, Royal Marsden NHS Foundation Trust/Institute of Cancer Research
- Action Medical Research
- National Institute for Health and Care Research
- University College London
- Great Ormond Street Hospital Charity
- Deutsche Forschungsgemeinschaft
- Great Ormond Street Hospital for Children
- Engineering and Physical Sciences Research Council (EP/G061521/1)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Health Sciences › Medicine › Pulmonary and Respiratory Medicine › Inhalation and Respiratory Drug Delivery
- Physical Sciences › Materials Science › Biomaterials › Nanoparticle-Based Drug Delivery
- Keywords
- epithelial sodium channel, cystic fibrosis, siRNA delivery, airway epithelial cells, liposomes, nebulization, air-liquid interface, sodium uptake, mucociliary function, airway surface liquid, gene silencing
- MeSH terms
- Cell Line, Cells, Cultured, Cystic Fibrosis, Epithelial Cells, Liposomes, Peptides, Transduction, Genetic, Transfection, Genetic Therapy, Gene Transfer Techniques, Microscopy, Confocal, Gene Silencing, RNA Interference, RNA, Small Interfering, Epithelial Sodium Channels, Nanoparticles, Gene Knockdown Techniques
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