Evidence map›Paper›PMID 42405279›Full record

ArticleMolecular therapy. Nucleic acids2026

Enhanced γ-globin reactivation and sickle cell correction through a repressor-to-activator motif switch in the

Anne Chalumeau, Panagiotis Antoniou, Maria Bou Dames, Pierre Martinucci, Elena Retana, Pragya Gupta, Mike Firth, Muralidhar Reddivari, Aathira Sarath Chandran, Jonathan S Yen and 4 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Nucleic acids, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed, 1 pooled it
–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

1 citing paper in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
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

14 authors.

Anne ChalumeauUniversité Paris Cité, Imagine Institute, Laboratory of Chromatin and Gene regulation During Development, INSERM UMR1163, 75015 Paris, France.
Panagiotis AntoniouGenome Engineering, Discovery Sciences, R&D, AstraZeneca, Gothenburg, Sweden.
Maria Bou DamesUniversité Paris Cité, Imagine Institute, Laboratory of Chromatin and Gene regulation During Development, INSERM UMR1163, 75015 Paris, France.
Pierre MartinucciUniversité Paris Cité, Imagine Institute, Laboratory of Chromatin and Gene regulation During Development, INSERM UMR1163, 75015 Paris, France.
Elena RetanaUniversité Paris Cité, Imagine Institute, Laboratory of Chromatin and Gene regulation During Development, INSERM UMR1163, 75015 Paris, France.
Pragya GuptaGenome Engineering, Discovery Sciences, R&D, AstraZeneca, Gothenburg, Sweden.
Mike FirthGenome Engineering, Discovery Sciences, R&D, AstraZeneca, Gothenburg, Sweden.
Muralidhar ReddivariDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
Aathira Sarath ChandranDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
Jonathan S YenDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
Martin PeterkaGenome Engineering, Discovery Sciences, R&D, AstraZeneca, Gothenburg, Sweden.
Marcello MarescaGenome Engineering, Discovery Sciences, R&D, AstraZeneca, Gothenburg, Sweden.
Mégane BrussonUniversité Paris Cité, Imagine Institute, Laboratory of Chromatin and Gene regulation During Development, INSERM UMR1163, 75015 Paris, France.
Annarita MiccioUniversité Paris Cité, Imagine Institute, Laboratory of Chromatin and Gene regulation During Development, INSERM UMR1163, 75015 Paris, France.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Sickle cell disease (SCD) is caused by the production of an abnormal adult hemoglobin that generates sickle-shaped red blood cells (RBCs). Transplantation of autologous genetically corrected hematopoietic stem/progenitor cells (HSPCs) represents a promising therapy. Persistent fetal hemoglobin expression improves SCD. Here, we engineered the fetal

Indexed as

activator binding sitefetal hemoglobingene editingMT: RNA/DNA editingprime editingsickle cell disease

Identifiers

PMID42405279
PMCPMC13330520

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.