Evidence map›Paper›PMID 33740872›Full record

ArticleHuman gene therapy2021

Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47

Andrea Schejtman, Winston Vetharoy, Uimook Choi, Christine Rivat, Narda Theobald, Giuseppa Piras, Diego Leon-Rico, Karen Buckland, Elena Armenteros-Monterroso, Sara Benedetti and 8 more

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

Article in Human gene therapy, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
1.0field-weighted citation impact, top 22% 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

3 citing papers in PubMed, 8 citations in OpenAlex.

  1. Article
  2. Review
  3. 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

18 authors at 5 institutions in 3 countries.

Andrea SchejtmanMolecular and Cellular Immunology Unit, UCL Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.
Winston VetharoyMolecular and Cellular Immunology Unit, UCL Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.
Uimook ChoiLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA.
Christine RivatGreat Ormond Street Hospital for Children, NHS Foundation Trust, London, United Kingdom.
Narda TheobaldLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA.
Giuseppa PirasMolecular and Cellular Immunology Unit, UCL Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.
Diego Leon-RicoMolecular and Cellular Immunology Unit, UCL Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.
Karen BucklandMolecular and Cellular Immunology Unit, UCL Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.
Elena Armenteros-MonterrosoMolecular and Cellular Immunology Unit, UCL Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.
Sara BenedettiMolecular and Cellular Immunology Unit, UCL Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.
Michael T AshworthDepartment of Histopathology, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, United Kingdom.
Michael RotheInstitute of Experimental Hematology, Hannover Medical School, Hannover, Germany.
Axel SchambachInstitute of Experimental Hematology, Hannover Medical School, Hannover, Germany.
Hubert Bobby GasparOrchard Therapeutics, London, United Kingdom.
Elizabeth M KangLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA.
Harry L MalechLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA.
Adrian J ThrasherMolecular and Cellular Immunology Unit, UCL Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.
Giorgia SantilliMolecular and Cellular Immunology Unit, UCL Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.
Great Ormond Street Hospital · GBNational Institutes of Health · USGreat Ormond Street Hospital for Children NHS Foundation Trust · GBBoston Children's Hospital · USMedizinische Hochschule Hannover · DE

Funding

Development and conduct of allogeneic stem cell transplant and autologous stem cell gene therapy for inherited immune deficienciesZIAAI000989 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI KANG, ELIZABETH · 2009 to 2025
$12.4M
Gene Therapy Basic Research to Treat Inherited Primary Immune DeficienciesZIAAI000644 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI MALECH, HARRY L · 2009 to 2025
$12.1M
Intramural NIH HHS ZIA AI000644Intramural NIH HHS ZIA AI000989Wellcome Trust 217112/Z/19/Z
6 · The paper itself

Abstract

Chronic granulomatous disease (CGD) is an inherited blood disorder of phagocytic cells that renders patients susceptible to infections and inflammation. A recent clinical trial of lentiviral gene therapy for the most frequent form of CGD, X-linked, has demonstrated stable correction over time, with no adverse events related to the gene therapy procedure. We have recently developed a parallel lentiviral vector for p47

Indexed as

Granulomatous Disease, ChronicAnimalsGenetic TherapyHumansMiceNADPH OxidasesTissue DistributionNADPH Oxidasesbiodistributionchronic granulomatous diseasegenotoxicitylentiviral gene therapy

Identifiers

PMID33740872
PMCPMC8575060
OpenAlexW3136725946

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.