Evidence map›Paper›PMID 31289207›Full record

ArticleHaematologica2020

Effective hematopoietic stem cell-based gene therapy in a murine model of hereditary pulmonary alveolar proteinosis.

Miriam Hetzel, Elena Lopez-Rodriguez, Adele Mucci, Ariane Hai Ha Nguyen, Takuji Suzuki, Kenjiro Shima, Theresa Buchegger, Sabine Dettmer, Thomas Rodt, Jens P Bankstahl and 7 more

Open access · goldAbstract read
In one paragraph

Article in Haematologica, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed
0.9field-weighted citation impact, top 23% 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

6 citing papers in PubMed, 11 citations in OpenAlex.

  1. Article
  2. A toxicology study ofMolecular therapy. Methods & clinical development · 2024
    Article
  3. Review
  4. Article
  5. Article
  6. 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 3 institutions in 3 countries.

Miriam HetzelInstitute of Experimental Hematology, Hannover Medical School, Hannover, Germany.
Elena Lopez-RodriguezInstitute of Functional and Applied Anatomy, Hannover Medical School, Hannover, Germany.
Adele MucciInstitute of Experimental Hematology, Hannover Medical School, Hannover, Germany.
Ariane Hai Ha NguyenInstitute of Experimental Hematology, Hannover Medical School, Hannover, Germany.
Takuji SuzukiTranslational Pulmonary Science Center, Division of Pulmonary Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
Kenjiro ShimaTranslational Pulmonary Science Center, Division of Pulmonary Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
Theresa BucheggerInstitute of Experimental Hematology, Hannover Medical School, Hannover, Germany.
Sabine DettmerDepartment of Radiology, Hannover Medical School, Hannover, Germany.
Thomas RodtDepartment of Radiology, Hannover Medical School, Hannover, Germany.
Jens P BankstahlDepartment of Nuclear Medicine, Hannover Medical School, Hannover, Germany.
Punam MalikDivision of Experimental Hematology and Cancer Biology, Cancer and Blood Disease Institute (CBDI), Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
Lars KnudsenInstitute of Functional and Applied Anatomy, Hannover Medical School, Hannover, Germany.
Axel SchambachInstitute of Experimental Hematology, Hannover Medical School, Hannover, Germany.
Gesine HansenDepartment of Pediatrics, Allergology, and Neonatology, Hannover Medical School, Hannover, Germany.
Bruce C TrapnellTranslational Pulmonary Science Center, Division of Pulmonary Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
Nico LachmannInstitute of Experimental Hematology, Hannover Medical School, Hannover, Germany.
Thomas MoritzInstitute of Experimental Hematology, Hannover Medical School, Hannover, Germany moritz.thomas@mh-hannover.de.
Medizinische Hochschule Hannover · DECincinnati Children's Hospital Medical Center · USJichi Medical University · JP

Funding

Role of GM-CSF in Myeloid Cell Function and Innate ImmunityR01HL085453 · NHLBI · CINCINNATI CHILDRENS HOSP MED CTR · PI TRAPNELL, BRUCE C · 2007 to 2023
$6.5M
Pulmonary Macrophage Transplantation for Pulmonary Alveolar ProteinosisR01HL118342 · NHLBI · CINCINNATI CHILDRENS HOSP MED CTR · PI TRAPNELL, BRUCE C, TRAPNELL, BRUCE COLSTON · 2014 to 2021
$4.5M
RLDC: Molecular Pathway-Driven Diagnostics & Therapeutics for Rare Lung DiseasesU54HL127672 · NHLBI · CINCINNATI CHILDRENS HOSP MED CTR · PI TRAPNELL, BRUCE C · 2014 to 2018
$3.7M
Genome edited iPS cell-derived macrophages as a novel lung cell therapyR01HL136721 · NHLBI · CINCINNATI CHILDRENS HOSP MED CTR · PI SUZUKI, TAKUJI · 2017 to 2020
$1.6M
NHLBI NIH HHS R01 HL085453NHLBI NIH HHS R01 HL118342NHLBI NIH HHS R01 HL136721NHLBI NIH HHS U54 HL127672
6 · The paper itself

Abstract

Hereditary pulmonary alveolar proteinosis due to GM-CSF receptor deficiency (herPAP) constitutes a life-threatening lung disease characterized by alveolar deposition of surfactant protein secondary to defective alveolar macrophage function. As current therapeutic options are primarily symptomatic, we have explored the potential of hematopoietic stem cell-based gene therapy. Using

Indexed as

Pulmonary Alveolar ProteinosisAnimalsDisease Models, AnimalGenetic TherapyHematopoietic Stem CellsMacrophages, AlveolarMice

Identifiers

PMID31289207
PMCPMC7109724
OpenAlexW2960876121

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
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.