Evidence map›Paper›PMID 34697091›Full record

ArticleThorax2022

Inhaled gene therapy of preclinical muco-obstructive lung diseases by nanoparticles capable of breaching the airway mucus barrier.

Namho Kim, Gijung Kwak, Jason Rodriguez, Alessandra Livraghi-Butrico, Xinyuan Zuo, Valentina Simon, Eric Han, Siddharth Kaup Shenoy, Nikhil Pandey, Marina Mazur and 6 more

Open access · greenAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Thorax, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.

0numbers the graph read from it
0cells of the map it votes in
10citing papers in PubMed
1.8field-weighted citation impact, top 14% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

10 citing papers in PubMed, 17 citations in OpenAlex.

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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

16 authors at 4 institutions in 1 country.

Namho KimCenter for Nanomedicine, Wilmer Eye Institute, Johns Hopkins Medicine, Baltimore, Maryland, USA.
Gijung KwakCenter for Nanomedicine, Wilmer Eye Institute, Johns Hopkins Medicine, Baltimore, Maryland, USA.ORCID 0000-0001-6908-9638
Jason RodriguezCenter for Nanomedicine, Wilmer Eye Institute, Johns Hopkins Medicine, Baltimore, Maryland, USA.
Alessandra Livraghi-ButricoMarisco Lung Institute and Cystic Fibrosis Research Center, University of North Carolina School of Medicine, Chapel Hill, North Carolina, USA.
Xinyuan ZuoDepartment of Chemical and Biomolecular Engineering, Johns Hopkins University, Whiting School of Engineering, Baltimore, Maryland, USA.
Valentina SimonCenter for Nanomedicine, Wilmer Eye Institute, Johns Hopkins Medicine, Baltimore, Maryland, USA.
Eric HanDepartment of Biomedical Engineering, Johns Hopkins University, Baltimore, Maryland, USA.
Siddharth Kaup ShenoyCenter for Nanomedicine, Wilmer Eye Institute, Johns Hopkins Medicine, Baltimore, Maryland, USA.
Nikhil PandeyDepartment of Neurosurgery, University of Maryland School of Medicine, Baltimore, Maryland, USA.
Marina MazurGregory Fleming James Cystic Fibrosis Research Center, The University of Alabama School of Medicine, Birmingham, Alabama, USA.
Susan E BirketGregory Fleming James Cystic Fibrosis Research Center, The University of Alabama School of Medicine, Birmingham, Alabama, USA.
Anthony KimDepartment of Neurosurgery, University of Maryland School of Medicine, Baltimore, Maryland, USA.
Steven M RoweGregory Fleming James Cystic Fibrosis Research Center, The University of Alabama School of Medicine, Birmingham, Alabama, USA.
Richard BoucherMarisco Lung Institute and Cystic Fibrosis Research Center, University of North Carolina School of Medicine, Chapel Hill, North Carolina, USA.
Justin HanesCenter for Nanomedicine, Wilmer Eye Institute, Johns Hopkins Medicine, Baltimore, Maryland, USA.
Jung Soo SukCenter for Nanomedicine, Wilmer Eye Institute, Johns Hopkins Medicine, Baltimore, Maryland, USA jsuk@jhmi.edu.
Johns Hopkins University · USUniversity of Alabama · USUniversity of Maryland, Baltimore · USUniversity of North Carolina at Chapel Hill · US

Funding

UAB CF Research and Translation Core CenterP30DK072482 · NIDDK · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI AMIT GAGGAR · 2007 to 2026
$23.0M
Wilmer Core Grant for Vision ResearchP30EY001765 · NEI · JOHNS HOPKINS UNIVERSITY · PI JEFFREY MUMM · 1985 to 2026
$22.1M
The Role of Airway Mucus in Infection and InflammationR01HL153079 · NHLBI · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI BIRKET, SUSAN ELIZABETH · 2021 to 2025
$2.2M
New Inhalation Therapy for COPDR01HL136617 · NHLBI · JOHNS HOPKINS UNIVERSITY · PI NEPTUNE, ENID R, SUK, JUNG SOO · 2017 to 2020
$1.6M
Biodegradable Mucus Penetrating DNA Nanoparticle for Gene Therapy of CFR01HL127413 · NHLBI · JOHNS HOPKINS UNIVERSITY · PI HANES, JUSTIN S., SUK, JUNG SOO · 2015 to 2018
$1.6M
NEI NIH HHS P30 EY001765NHLBI NIH HHS R01 HL127413NHLBI NIH HHS R01 HL136617NHLBI NIH HHS R01 HL153079NIDDK NIH HHS P30 DK072482
6 · The paper itself

Abstract

introductionInhaled gene therapy of muco-obstructive lung diseases requires a strategy to achieve therapeutically relevant gene transfer to airway epithelium covered by particularly dehydrated and condensed mucus gel layer. Here, we introduce a synthetic DNA-loaded mucus-penetrating particle (DNA-MPP) capable of providing safe, widespread and robust transgene expression in in vivo and in vitro models of muco-obstructive lung diseases.

methodsWe investigated the ability of DNA-MPP to mediate reporter and/or therapeutic transgene expression in lung airways of a transgenic mouse model of muco-obstructive lung diseases (ie,

resultsDNA-MPP exhibited marked greater reporter transgene expression compared with a mucus-impermeable formulation in in vivo and in vitro models of muco-obstructive lung diseases. DNA-MPP carrying ENaC-silencing plasmids provided efficient downregulation of ENaC and reduction of mucus burden in the lungs of DISCUSSION: DNA-MPP constitutes one of the rare gene delivery systems providing therapeutically meaningful gene transfer efficacy in highly relevant in vivo and in vitro models of muco-obstructive lung diseases due to its unique ability to efficiently penetrate airway mucus.

Indexed as

Lung Diseases, ObstructiveNanoparticlesAnimalsDNAGenetic TherapyHumansLungMiceMucusDNAcystic fibrosis

Identifiers

PMID34697091
PMCPMC9129924
OpenAlexW3208483117

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.