Evidence map›Paper›PMID 42181695›Full record

ArticleMolecular therapy. Nucleic acids2026

Modeling and correction of SCID-X1 using CRISPR-Cas9 homology-directed repair in human HSPCs.

Orli Knop, Nimrod Ben Haim, Nechama Kalter, Michael Rosenberg, Dor Breier, Katia Baider, Yu Nee Lee, Etai Adam, Ido Somekh, Arnon Nagler and 2 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. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing 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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

12 authors.

Orli KnopInstitute of Nanotechnology and Advanced Materials, The Mina and Everard Goodman Faculty of Life Sciences, Bar-Ilan University, Ramat-Gan, Israel.
Nimrod Ben HaimInstitute of Nanotechnology and Advanced Materials, The Mina and Everard Goodman Faculty of Life Sciences, Bar-Ilan University, Ramat-Gan, Israel.
Nechama KalterInstitute of Nanotechnology and Advanced Materials, The Mina and Everard Goodman Faculty of Life Sciences, Bar-Ilan University, Ramat-Gan, Israel.
Michael RosenbergInstitute of Nanotechnology and Advanced Materials, The Mina and Everard Goodman Faculty of Life Sciences, Bar-Ilan University, Ramat-Gan, Israel.
Dor BreierInstitute of Nanotechnology and Advanced Materials, The Mina and Everard Goodman Faculty of Life Sciences, Bar-Ilan University, Ramat-Gan, Israel.
Katia BaiderThe Division of Hematology and Bone Marrow Transplantation, Chaim Sheba Medical Center, Tel-Hashomer, Ramat Gan, Israel.
Yu Nee LeeSackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.
Etai AdamDepartment of Pediatric Hematology, Oncology, and Bone Marrow Transplant, Edmond and Lily Safra Children's Hospital, Sheba Medical Center, Ramat Gan, Israel.
Ido SomekhPediatric Department A and the Immunology Service, Jeffrey Modell Foundation Center, Edmond and Lily Safra Children's Hospital, Sheba Medical Center, Ramat Gan, Israel.
Arnon NaglerThe Division of Hematology and Bone Marrow Transplantation, Chaim Sheba Medical Center, Tel-Hashomer, Ramat Gan, Israel.
Raz SomechSackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.
Ayal HendelInstitute of Nanotechnology and Advanced Materials, The Mina and Everard Goodman Faculty of Life Sciences, Bar-Ilan University, Ramat-Gan, Israel.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

X-linked severe combined immunodeficiency (SCID-X1) is a severe primary immunodeficiency caused by mutations in the

Indexed as

CRISPR-Cas9gene replacementgenome editingHSPCsIL2RGMT: RNA/DNA EditingrAAV6SCID-X1

Identifiers

PMID42181695
PMCPMC13191621

What OpenQuestion holds

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Registered trials

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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.