Cited by 210RNA Interference and Gene DeliveryInhalation and Respiratory Drug DeliveryNanoparticle-Based Drug Delivery

Highly compacted biodegradable DNA nanoparticles capable of overcoming the mucus barrier for inhaled lung gene therapy

Open access
Mastorakos, Panagiotis, da Silva, Adriana L., Chisholm, Jane, Song, Eric, Choi, Won Kyu, Boyle, Michael P., Morales, Marcelo M., Hanes, Justin +1 more·2015·Proceedings of the National Academy of Sciences

Abstract

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.

Integrity signals

Registry

Crossref · 10.1073/pnas.1502281112

Cites

66 of 66 references verified0 citation errors · 0 not checkable

Statistics

Recomputing p-values needs the paper’s full text.

Claims the paper makes

We couldn’t retrieve this paper’s full text, so its claims and self-check weren’t run. The registry and citation checks still apply.

Claims other papers make about this one

Each is checked against this paper’s own text: does it say what the citing paper says it does?

  • Biodegradable DNA NPs have shown potential for inhaled lung gene therapy, capable of overcoming mucus barrier of lung airways.

    “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”

Cites

Flagged first, then by citations

Cited by

Sorted by citations

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)
Venue
Proceedings of the National Academy of Sciences
Published
2015-06-29
Volume
112
Issue
28
Pages
8720-8725
ISSN
  • 0027-8424 (ISSN-L)
  • 1091-6490
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
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

About these checks

Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.

Statistics and arithmetic are recomputed, not judged.

Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.

What counts as a claim ›How we measure confidence ›How we check this ›

Are you an author, or do you see a problem with this page? Request a review