A Two-Pronged Pulmonary Gene Delivery Strategy: A Surface-Modified Fullerene Nanoparticle and a Hypotonic Vehicle.

Chronic Disease Epoxy Compounds / chemistry Fullerenes / chemistry Gene Transfer Techniques Humans Lung Diseases / metabolism Nanoparticles / chemistry
RVD effect fullerene gene delivery hypotonic vehicles inhalation

Journal

Angewandte Chemie (International ed. in English)
ISSN: 1521-3773
Titre abrégé: Angew Chem Int Ed Engl
Pays: Germany
ID NLM: 0370543

Informations de publication

Date de publication:
05 07 2021
Historique:
revised: 30 03 2021
received: 03 02 2021
pubmed: 16 4 2021
medline: 16 9 2021
entrez: 15 4 2021
Statut: ppublish

Résumé

Inhaled gene therapy poses a unique potential of curing chronic lung diseases, which are currently managed primarily by symptomatic treatments. However, it has been challenging to achieve therapeutically relevant gene transfer efficacy in the lung due to the presence of numerous biological delivery barriers. Here, we introduce a simple approach that overcomes both extracellular and cellular barriers to enhance gene transfer efficacy in the lung in vivo. We endowed tetra(piperazino)fullerene epoxide (TPFE)-based nanoparticles with non-adhesive surface polyethylene glycol (PEG) coatings, thereby enabling the nanoparticles to cross the airway mucus gel layer and avoid phagocytic uptake by alveolar macrophages. In parallel, we utilized a hypotonic vehicle to facilitate endocytic uptake of the PEGylated nanoparticles by lung parenchymal cells via the osmotically driven regulatory volume decrease (RVD) mechanism. We demonstrate that this two-pronged delivery strategy provides safe, wide-spread and high-level transgene expression in the lungs of both healthy mice and mice with chronic lung diseases characterized by reinforced delivery barriers.

Identifiants

DOI: 10.1002/anie.202101732 PMID: 33855792 PMC: PMC8238871
pubmed: 33855792
doi: 10.1002/anie.202101732
pmc: PMC8238871
mid: NIHMS1707644
doi:

Substances chimiques

Epoxy Compounds 0
Fullerenes 0

Types de publication

Journal Article Research Support, N.I.H., Extramural Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

15225-15229

Subventions

Organisme : NEI NIH HHS
ID : P30 EY001765
Pays : United States
Organisme : NHLBI NIH HHS
ID : R01 HL136617
Pays : United States

Informations de copyright

© 2021 The Authors. Angewandte Chemie International Edition published by Wiley-VCH GmbH.

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Auteurs

Daiqin Chen (D)

The Center for Nanomedicine at the Wilmer Eye Institute, Johns Hopkins University School of Medicine, Johns Hopkins, Baltimore, MD, USA.
Department of Ophthalmology, Johns Hopkins University, Baltimore, MD, USA.

Shuai Liu (S)

Key Laboratory of Molecular Nanostructure and Nanotechnology, Institute of Chemistry, Chinese Academy of Sciences, Zhongguancun North First Street 2, Beijing, PR China.

Dinghao Chen (D)

The Center for Nanomedicine at the Wilmer Eye Institute, Johns Hopkins University School of Medicine, Johns Hopkins, Baltimore, MD, USA.
Department of Chemical and Biomolecular Engineering, Whiting School of Engineering, Johns Hopkins University, Baltimore, MD, USA.

Jinhao Liu (J)

The Center for Nanomedicine at the Wilmer Eye Institute, Johns Hopkins University School of Medicine, Johns Hopkins, Baltimore, MD, USA.
Department of Chemical and Biomolecular Engineering, Whiting School of Engineering, Johns Hopkins University, Baltimore, MD, USA.

Jerry Wu (J)

The Center for Nanomedicine at the Wilmer Eye Institute, Johns Hopkins University School of Medicine, Johns Hopkins, Baltimore, MD, USA.

Han Wang (H)

Department of Chemical and Biomolecular Engineering, Whiting School of Engineering, Johns Hopkins University, Baltimore, MD, USA.

Yun Su (Y)

The Center for Nanomedicine at the Wilmer Eye Institute, Johns Hopkins University School of Medicine, Johns Hopkins, Baltimore, MD, USA.
Department of Ophthalmology, Johns Hopkins University, Baltimore, MD, USA.

Gijung Kwak (G)

The Center for Nanomedicine at the Wilmer Eye Institute, Johns Hopkins University School of Medicine, Johns Hopkins, Baltimore, MD, USA.
Department of Ophthalmology, Johns Hopkins University, Baltimore, MD, USA.

Xinyuan Zuo (X)

The Center for Nanomedicine at the Wilmer Eye Institute, Johns Hopkins University School of Medicine, Johns Hopkins, Baltimore, MD, USA.
Department of Chemical and Biomolecular Engineering, Whiting School of Engineering, Johns Hopkins University, Baltimore, MD, USA.

Divya Rao (D)

The Center for Nanomedicine at the Wilmer Eye Institute, Johns Hopkins University School of Medicine, Johns Hopkins, Baltimore, MD, USA.
Department of Chemical and Biomolecular Engineering, Whiting School of Engineering, Johns Hopkins University, Baltimore, MD, USA.

Honggang Cui (H)

Department of Chemical and Biomolecular Engineering, Whiting School of Engineering, Johns Hopkins University, Baltimore, MD, USA.

Chunying Shu (C)

Key Laboratory of Molecular Nanostructure and Nanotechnology, Institute of Chemistry, Chinese Academy of Sciences, Zhongguancun North First Street 2, Beijing, PR China.

Jung Soo Suk (JS)

The Center for Nanomedicine at the Wilmer Eye Institute, Johns Hopkins University School of Medicine, Johns Hopkins, Baltimore, MD, USA.
Department of Ophthalmology, Johns Hopkins University, Baltimore, MD, USA.
Department of Chemical and Biomolecular Engineering, Whiting School of Engineering, Johns Hopkins University, Baltimore, MD, USA.
ORCID: 0000-0002-2437-0001

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Classifications MeSH

questionsmedicales.fr › États, signes et symptômes pathologiques › Processus pathologiques › Caractéristiques de la maladie › Maladie chronique › A Two-Pronged Pulmonary Gene Delivery...
questionsmedicales.fr › Composés chimiques organiques › Éthers › Composés époxy › A Two-Pronged Pulmonary Gene Delivery...
questionsmedicales.fr › Technologie, industrie et agriculture › Produits manufacturés › Nanostructures › Nanoparticules › Nanosphères › Fullerènes › A Two-Pronged Pulmonary Gene Delivery...
questionsmedicales.fr › Techniques d'investigation › Techniques génétiques › Techniques de transfert de gènes › A Two-Pronged Pulmonary Gene Delivery...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › A Two-Pronged Pulmonary Gene Delivery...
questionsmedicales.fr › Maladies de l'appareil respiratoire › Maladies pulmonaires › A Two-Pronged Pulmonary Gene Delivery...
questionsmedicales.fr › Technologie, industrie et agriculture › Produits manufacturés › Nanostructures › Nanoparticules › A Two-Pronged Pulmonary Gene Delivery...