Evidence map›Paper›PMID 40107272›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2025

Safety and efficacy of gene therapy for RAG1-deficient SCID.

Frank J T Staal, Karin Pike-Overzet, Sander de Kivit, Lisa Ott de Bruin, Lucia Mamede, Martine Pergent, Johan Prevot, Michael Rothe, Axel Schambach, Arjan Lankester

Abstract readLetter
In one paragraph

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2025. 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
–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

2 citing papers in PubMed.

  1. Article
  2. Response to: Safety and efficacy considerations of HSC-based gene therapy for RAG1-deficient SCID.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    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

10 authors.

Frank J T StaalLeiden University Medical Center, Leiden, the Netherlands. Electronic address: f.j.t.staal@lumc.nl.
Karin Pike-OverzetLeiden University Medical Center, Leiden, the Netherlands.
Sander de KivitLeiden University Medical Center, Leiden, the Netherlands.
Lisa Ott de BruinLeiden University Medical Center, Leiden, the Netherlands.
Lucia MamedeInternational Patient Organization for Primary Immune Deficiencies (IPOPI), Brussels, Belgium.
Martine PergentInternational Patient Organization for Primary Immune Deficiencies (IPOPI), Brussels, Belgium.
Johan PrevotInternational Patient Organization for Primary Immune Deficiencies (IPOPI), Brussels, Belgium.
Michael RotheInstitute of Experimental Hematology, Hannover Medical School, Hannover, Germany.
Axel SchambachInstitute of Experimental Hematology, Hannover Medical School, Hannover, Germany.
Arjan LankesterLeiden University Medical Center, Leiden, the Netherlands.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

PubMed holds no abstract for this paper.

Identifiers

PMID40107272
PMCPMC12126822

What OpenQuestion holds

Textmetadata
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.