Evidence map›Paper›PMID 39062069›Full record

ArticleBiomedicines2024

Restoration of T and B Cell Differentiation after RAG1 Gene Transfer in Human RAG1 Defective Hematopoietic Stem Cells.

Nataël Sorel, Francisco Díaz-Pascual, Boris Bessot, Hanem Sadek, Chloé Mollet, Myriam Chouteau, Marco Zahn, Irene Gil-Farina, Parisa Tajer, Marja van Eggermond and 8 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Biomedicines, 2024. 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
–field-weighted citation impact
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.

  1. Trial
  2. Article
  3. Ex vivo modification of hematopoietic stem and progenitor cells for gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    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

18 authors.

Nataël SorelHuman Lymphohematopoiesis Laboratory, Université Paris Cité, Imagine Institute, INSERM UMR 1163, 75015 Paris, France.ORCID 0009-0000-8202-0600
Francisco Díaz-PascualProtaGene CGT GmbH, Im Neuenheimer Feld 582, 69120 Heidelberg, Germany.
Boris BessotBiotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM, 75015 Paris, France.
Hanem SadekHuman Lymphohematopoiesis Laboratory, Université Paris Cité, Imagine Institute, INSERM UMR 1163, 75015 Paris, France.
Chloé MolletBiotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM, 75015 Paris, France.ORCID 0009-0007-7569-9441
Myriam ChouteauHuman Lymphohematopoiesis Laboratory, Université Paris Cité, Imagine Institute, INSERM UMR 1163, 75015 Paris, France.
Marco ZahnProtaGene CGT GmbH, Im Neuenheimer Feld 582, 69120 Heidelberg, Germany.
Irene Gil-FarinaProtaGene CGT GmbH, Im Neuenheimer Feld 582, 69120 Heidelberg, Germany.
Parisa TajerDepartment of Immunohematology and Blood Transfusion, L3-Q Leiden University Medical Center, 2333 ZA Leiden, The Netherlands.
Marja van EggermondDepartment of Immunohematology and Blood Transfusion, L3-Q Leiden University Medical Center, 2333 ZA Leiden, The Netherlands.
Dagmar BerghuisDepartment of Pediatrics, Leiden University Medical Center, 2333 ZA Leiden, The Netherlands.
Arjan C LankesterDepartment of Pediatrics, Leiden University Medical Center, 2333 ZA Leiden, The Netherlands.
Isabelle AndréHuman Lymphohematopoiesis Laboratory, Université Paris Cité, Imagine Institute, INSERM UMR 1163, 75015 Paris, France.
Richard GabrielProtaGene CGT GmbH, Im Neuenheimer Feld 582, 69120 Heidelberg, Germany.ORCID 0009-0006-8123-001X
Marina CavazzanaBiotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM, 75015 Paris, France.ORCID 0000-0002-0264-0891
Kasrin Pike-OverzetBiotherapy Department, Necker-Enfants Malades Hospital, AP-HP, 75015 Paris, France.
Frank J T StaalDepartment of Immunohematology and Blood Transfusion, L3-Q Leiden University Medical Center, 2333 ZA Leiden, The Netherlands.ORCID 0000-0003-1588-8519
Chantal Lagresle-PeyrouHuman Lymphohematopoiesis Laboratory, Université Paris Cité, Imagine Institute, INSERM UMR 1163, 75015 Paris, France.ORCID 0000-0002-2216-6453

Funding

European Commission 755170-2Novo Nordisk Foundation NNF21CC0073729
6 · The paper itself

Abstract

Recombinase-activating gene (RAG)-deficient SCID patients lack B and T lymphocytes due to the inability to rearrange immunoglobulin and T cell receptor genes. The two

Indexed as

gene therapy (GT)recombination activating gene 1 (RAG1)severe combined immunodeficiency (SCID)

Identifiers

PMID39062069
PMCPMC11275127

What Socratic 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 Socratic graph.