Evidence map›Paper›PMID 31543470›Full record

ArticleStem cell reports2019

Targeted Repair of p47-CGD in iPSCs by CRISPR/Cas9: Functional Correction without Cleavage in the Highly Homologous Pseudogenes.

Denise Klatt, Erica Cheng, Friederike Philipp, Anton Selich, Julia Dahlke, Reinhold E Schmidt, Juliane W Schott, Hildegard Büning, Dirk Hoffmann, Adrian J Thrasher and 1 more

Open access · goldAbstract read
In one paragraph

Article in Stem cell reports, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers.

0numbers the graph read from it
0cells of the map it votes in
23citing papers in PubMed
1.5field-weighted citation impact, top 17% 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

23 citing papers in PubMed, 27 citations in OpenAlex.

  1. Article
  2. Review
  3. Article
  4. Review
  5. Article
  6. Targeted knock-in ofMolecular therapy. Nucleic acids · 2024
    Article
  7. Review
  8. Review
  9. A Model of iPSC-Derived Macrophages withInternational journal of molecular sciences · 2023
    Article
  10. Article
  11. Review
  12. Review
  13. Frontiers in immunology · 2022
    Review
  14. A missense variant inOpen life sciences · 2022
    Article
  15. Review
  16. Review
  17. Chronic Granulomatous Disease: a Comprehensive Review.Clinical reviews in allergy & immunology · 2021
    Review
  18. Article
  19. Review
  20. Genome Editing for Rare Diseases.Current stem cell reports · 2020
    Article
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

11 authors at 3 institutions in 3 countries.

Denise KlattInstitute of Experimental Hematology, Hannover Medical School, Carl-Neuberg-Strasse 1, 30625 Hannover, Germany; REBIRTH Cluster of Excellence, Hannover Medical School, 30625 Hannover, Germany.
Erica ChengInstitute of Experimental Hematology, Hannover Medical School, Carl-Neuberg-Strasse 1, 30625 Hannover, Germany; REBIRTH Cluster of Excellence, Hannover Medical School, 30625 Hannover, Germany.
Friederike PhilippInstitute of Experimental Hematology, Hannover Medical School, Carl-Neuberg-Strasse 1, 30625 Hannover, Germany; REBIRTH Cluster of Excellence, Hannover Medical School, 30625 Hannover, Germany; Fraunhofer Institute for Toxicology and Experimental Medicine, 30625 Hannover, Germany.
Anton SelichInstitute of Experimental Hematology, Hannover Medical School, Carl-Neuberg-Strasse 1, 30625 Hannover, Germany.
Julia DahlkeInstitute of Experimental Hematology, Hannover Medical School, Carl-Neuberg-Strasse 1, 30625 Hannover, Germany; REBIRTH Cluster of Excellence, Hannover Medical School, 30625 Hannover, Germany.
Reinhold E SchmidtDepartment of Immunology and Rheumatology, Hannover Medical School, 30625 Hannover, Germany.
Juliane W SchottInstitute of Experimental Hematology, Hannover Medical School, Carl-Neuberg-Strasse 1, 30625 Hannover, Germany.
Hildegard BüningInstitute of Experimental Hematology, Hannover Medical School, Carl-Neuberg-Strasse 1, 30625 Hannover, Germany; REBIRTH Cluster of Excellence, Hannover Medical School, 30625 Hannover, Germany.
Dirk HoffmannInstitute of Experimental Hematology, Hannover Medical School, Carl-Neuberg-Strasse 1, 30625 Hannover, Germany; REBIRTH Cluster of Excellence, Hannover Medical School, 30625 Hannover, Germany.
Adrian J ThrasherInfection, Immunity and Inflammation Program, Molecular and Cellular Immunology Section, UCL Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK; Great Ormond Street Hospital NHS Foundation Trust, London WC1N 1EH, UK.
Axel SchambachInstitute of Experimental Hematology, Hannover Medical School, Carl-Neuberg-Strasse 1, 30625 Hannover, Germany; REBIRTH Cluster of Excellence, Hannover Medical School, 30625 Hannover, Germany; Division of Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA 02115, USA. Electronic address: schambach.axel@mh-hannover.de.
Medizinische Hochschule Hannover · DEGreat Ormond Street Hospital for Children NHS Foundation Trust · GBHarvard University · US

Funding

Wellcome TrustWellcome Trust 104807/Z/14/Z
6 · The paper itself

Abstract

Mutations in the NADPH oxidase, which is crucial for the respiratory burst in phagocytes, result in chronic granulomatous disease (CGD). The only curative treatment option for CGD patients, who suffer from severe infections, is allogeneic bone marrow transplantation. Over 90% of patients with mutations in the p47

Indexed as

CRISPR-Cas SystemsGene EditingEnzyme ActivationGene ExpressionGene TargetingGenetic LociGranulocytesGranulomatous Disease, ChronicHumansInduced Pluripotent Stem CellsIntronsMacrophagesNADPH OxidasesPhagocytosisPseudogenesSequence HomologyNADPH Oxidasesneutrophil cytosolic factor 1chronic granulomatous disease (CGD)CRISPR/Cas9gene editinghuman induced pluripotent stem cellsNADPH oxidaseNCF1p47phoxpseudogenes

Identifiers

PMID31543470
PMCPMC6829751
OpenAlexW2974703082

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.