The DOI matches its Crossref record; 66 of the 66 references it cites were found in a registry or an index.
Highly compacted biodegradable DNA nanoparticles capable of overcoming the mucus barrier for inhaled lung gene therapy
Open accessAbstract
Significance: Therapeutically relevant lung gene therapy is yet to be achieved. We introduce a highly translatable gene delivery platform for inhaled gene therapy based on state-of-the-art biodegradable polymers, poly(β-amino esters). The newly designed system is capable of overcoming challenging biological barriers, thereby providing robust transgene expression throughout the entire luminal surface of mouse lungs. Moreover, it provides markedly greater overall transgene expression in vivo compared with gold standard platforms, including a clinically tested system. The clinical relevance is further underscored by the excellent safety profile as well as long-term and consistent transgene expression achieved following a single and repeated administrations, respectively.
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“In recent times, biodegradable DNA NPs have shown potential for inhaled lung gene therapy, which is capable of overcoming the mucus barrier of lung airways.183”
From Targeted therapy in chronic diseases using nanomaterial-based drug delivery vehicles (2019) · cites this paper as [183]
Citations
Cites
Nonviral vectors for gene delivery
Mintzer, M. A., & Simanek, E. E. (2009) · Chemical Reviews · doi:10.1021/cr800409e · Cited by 2,366
Mucus-penetrating nanoparticles for drug and gene delivery to mucosal tissues
Lai, S. K., Wang, Y.-Y., & Hanes, J. S. (2008) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2008.11.002 · Cited by 1,863
Self-citationCone, R. A. (2008) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2008.09.008 · Cited by 1,459
Degradable poly (β-amino esters): synthesis, characterization, and self-assembly with plasmid DNA
Lynn, D. M., & Langer, R. (2000) · Journal of the American Chemical Society · doi:10.1021/ja0015388 · Cited by 924
Mishra, S., Webster, P. C., & Davis, M. E. (2004) · European Journal of Cell Biology · doi:10.1078/0171-9335-00363 · Cited by 740
Cell number and cell characteristics of the normal human lung.
Crapo, J. D., Barry, B. E., Gehr, P., Bachofen, M., & Weibel, E. R. (1982) · PubMed · doi:10.1164/arrd.1982.125.6.740 · Cited by 730
Immune responses to gene therapy vectors: influence on vector function and effector mechanisms
Bessis, N., Garcia-Cozar, F. J., & Boissier, M. (2004) · Gene Therapy · doi:10.1038/sj.gt.3302364 · Cited by 653
New Drugs for Rheumatoid Arthritis
Olsen, N. J., & Stein, C. (2004) · New England Journal of Medicine · doi:10.1056/nejmra032906 · Cited by 632
A combinatorial polymer library approach yields insight into nonviral gene delivery
Green, J. J., Langer, R., & Anderson, D. G. (2008) · Accounts of Chemical Research · doi:10.1021/ar7002336 · Cited by 579
Nanoparticle diffusion in respiratory mucus from humans without lung disease
Schuster, B. S., Suk, J. S., Woodworth, G. F., & Hanes, J. S. (2013) · Biomaterials · doi:10.1016/j.biomaterials.2013.01.064 · Cited by 478
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Cited by
Sorted by citations
PEGylation as a strategy for improving nanoparticle-based drug and gene delivery
Suk, J. S., Xu, Q., Kim, N., Hanes, J., & Ensign, L. M. (2016) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2015.09.012 · Cited by 4,523
Self-citationThe current state and future directions of RNAi-based therapeutics
Setten, R. L., Rossi, J. J., & Han, S.-P. (2019) · Nature Reviews Drug Discovery · doi:10.1038/s41573-019-0017-4 · Cited by 1,443
Drug delivery systems for RNA therapeutics
Paunovska, K., Loughrey, D., & Dahlman, J. E. (2022) · Nature Reviews Genetics · doi:10.1038/s41576-021-00439-4 · Cited by 1,349
Targeted therapy in chronic diseases using nanomaterial-based drug delivery vehicles
Singh, A. P., Biswas, A., Shukla, A., & Maiti, P. (2019) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-019-0068-3 · Cited by 645
Physiology and pathophysiology of human airway mucus
Hill, D. B., Button, B. M., Rubinstein, M., & Boucher, R. C. (2022) · Physiological Reviews · doi:10.1152/physrev.00004.2021 · Cited by 406
Metadata
- Authors
- Mastorakos, P., da Silva, A. L., Chisholm, J., Song, E., Choi, W. K., Boyle, M. P., Morales, M. M., Hanes, J., & Suk, J. S.
- Affiliations
- Johns Hopkins University (US)
- Laboratory of Cellular and Molecular Physiology, Carlos Chagas Filho Institute of Biophysics, Federal University of Rio de Janeiro, Rio de Janeiro, RJ 21941902, Brazil;
- Universidade Federal do Rio de Janeiro (BR)
- ORCID
- Venue
- Proceedings of the National Academy of Sciences
- Publisher
- National Academy of Sciences
- Published
- 2015-06-29
- Volume
- 112
- Issue
- 28
- Pages
- 8720-8725
- ISSN
- 0027-8424 (ISSN-L)
- 1091-6490
- PMC
- PMC4507234
- OpenAlex
- W2149239077
- Cited by
- 210
- References
- 66
- Citations by year
- 2026: 15 · 2025: 13 · 2024: 20 · 2023: 20 · 2022: 17 · 2021: 24 · 2020: 22 · 2019: 21 · 2018: 20 · 2017: 20 · 2016: 15 · 2015: 3
- FWCI
- 8.40
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Proceedings of the National Academy of Sciences (publishedVersion)
- Europe PMC (PubMed Central) (publishedVersion)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- Funders
- HHS | National Institutes of Health (P01 HL51811, R01 HL127413)
- Cystic Fibrosis Foundation (HANES07XX0, HANES15G0)
- 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
- DNA nanoparticles, pulmonary gene therapy, mucus barrier, poly(β-amino ester), biodegradable polymers, transgene expression, nanoparticle stability, intratracheal administration, nanoparticle toxicity
- MeSH terms
- Administration, Inhalation, Animals, Cystic Fibrosis, DNA, Mucus, Genetic Therapy, Mice, Nanoparticles
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