Evidence map›Paper›PMID 35573048›Full record

ArticleMolecular therapy. Methods & clinical development2022

Correction of a chronic pulmonary disease through lentiviral vector-mediated protein expression.

Helena Lund-Palau, Claudia Ivette Juarez-Molina, Cuixiang Meng, Anushka Bhargava, Aikaterini Pilou, Kiran Aziz, Nora Clarke, Naoko Atsumi, Ali Ashek, Michael R Wilson and 7 more

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

Article in Molecular therapy. Methods & clinical development, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
0.2field-weighted citation impact, top 53% 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

2 citing papers in PubMed, 1 citations in OpenAlex.

  1. Article
  2. Progress in Respiratory Gene Therapy.Human gene therapy · 2022
    Review
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

17 authors at 4 institutions in 1 country.

Helena Lund-PalauNational Heart and Lung Institute, Gene Therapy Group, Imperial College London, Faculty of Medicine, Manresa Road, London SW3 6LR, UK.
Claudia Ivette Juarez-MolinaNational Heart and Lung Institute, Gene Therapy Group, Imperial College London, Faculty of Medicine, Manresa Road, London SW3 6LR, UK.
Cuixiang MengNational Heart and Lung Institute, Gene Therapy Group, Imperial College London, Faculty of Medicine, Manresa Road, London SW3 6LR, UK.
Anushka BhargavaNational Heart and Lung Institute, Gene Therapy Group, Imperial College London, Faculty of Medicine, Manresa Road, London SW3 6LR, UK.
Aikaterini PilouNational Heart and Lung Institute, Gene Therapy Group, Imperial College London, Faculty of Medicine, Manresa Road, London SW3 6LR, UK.
Kiran AzizNational Heart and Lung Institute, Gene Therapy Group, Imperial College London, Faculty of Medicine, Manresa Road, London SW3 6LR, UK.
Nora ClarkeNational Heart and Lung Institute, Gene Therapy Group, Imperial College London, Faculty of Medicine, Manresa Road, London SW3 6LR, UK.
Naoko AtsumiNational Heart and Lung Institute, Gene Therapy Group, Imperial College London, Faculty of Medicine, Manresa Road, London SW3 6LR, UK.
Ali AshekNational Heart and Lung Institute, Gene Therapy Group, Imperial College London, Faculty of Medicine, Manresa Road, London SW3 6LR, UK.
Michael R WilsonDivision of Anesthetics, Pain Medicine, and Intensive Care, Department of Surgery and Cancer, Imperial College London, London, UK.
Masao TakataDivision of Anesthetics, Pain Medicine, and Intensive Care, Department of Surgery and Cancer, Imperial College London, London, UK.
Simon PadleyRoyal Brompton Hospital & Harefield Hospitals, London, UK.
Deborah R GillRadcliffe Department of Medicine, University of Oxford, Oxford, UK.
Stephen C HydeRadcliffe Department of Medicine, University of Oxford, Oxford, UK.
Cliff MorganRoyal Brompton Hospital & Harefield Hospitals, London, UK.
Eric W F W AltonNational Heart and Lung Institute, Gene Therapy Group, Imperial College London, Faculty of Medicine, Manresa Road, London SW3 6LR, UK.
Uta GriesenbachNational Heart and Lung Institute, Gene Therapy Group, Imperial College London, Faculty of Medicine, Manresa Road, London SW3 6LR, UK.
Cell and Gene Therapy Catapult · GBImperial College London · GBHarefield Hospital · GBUniversity of Oxford · GB

Funding

Wellcome Trust
6 · The paper itself

Abstract

We developed a novel lentiviral vector, pseudotyped with the F and HN proteins from Sendai virus (rSIV.F/HN), that produces long-lasting, high-efficiency transduction of the respiratory epithelium. Here we addressed whether this platform technology can secrete sufficient levels of a therapeutic protein into the lungs to ameliorate a fatal pulmonary disease as an example of its translational capability. Pulmonary alveolar proteinosis (PAP) results from alveolar granulocyte-macrophage colony-stimulating factor (GM-CSF) insufficiency, resulting in abnormal surfactant homeostasis and consequent ventilatory problems. Lungs of GM-CSF knockout mice were transduced with a single dose of rSIV.F/HN-expressing murine GM-CSF (mGM-CSF; 1e5-92e7 transduction units [TU]/mouse); mGM-CSF expression was dose related and persisted for at least 11 months. PAP disease biomarkers were rapidly and persistently corrected, but we noted a narrow toxicity/efficacy window. rSIV.F/HN may be a useful platform technology to deliver therapeutic proteins for lung diseases requiring long-lasting and stable expression of secreted proteins.

Indexed as

alveolar macrophageautoimmune pulmonary alveolar proteinosisgene therapyGM-CSFgranulocyte-macrophage colony-stimulating factorlentiviral vectorlung gene therapyPAPrSIV.F/HNsurfactant protein

Identifiers

PMID35573048
PMCPMC9065048
OpenAlexW4224068316

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.