Evidence map›Paper›PMID 40012884›Full record

ArticleMolecular therapy. Methods & clinical development2024

DNA-PK inhibition enhances gene editing efficiency in HSPCs for CRISPR-based treatment of X-linked hyper IgM syndrome.

Cole M Pugliano, Mason Berger, Roslyn M Ray, Kai Sapkos, Betty Wu, Aidan Laird, Yidian Ye, Daniel Thomson, M Quinn DeGottardi, Iram F Khan and 6 more

Abstract read
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
7citing 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

7 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. CRISPR for cystic fibrosis: Advances and insights from a systematic review.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Pooled it
  2. Article
  3. Review
  4. Article
  5. Advancing AAV technology-From promise to product.Molecular therapy. Advances · 2026
    Article
  6. Precision T cell correction platform for inborn errors of immunity.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Article
  7. 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

16 authors.

Cole M PuglianoCenter for Immunity and Immunotherapies and the Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA.
Mason BergerCenter for Immunity and Immunotherapies and the Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA.
Roslyn M RayGene Therapy Research, CSL Behring, Pasadena, CA 91101, USA.
Kai SapkosGene Therapy Research, CSL Behring, Pasadena, CA 91101, USA.
Betty WuGene Therapy Research, CSL Behring, Pasadena, CA 91101, USA.
Aidan LairdCenter for Immunity and Immunotherapies and the Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA.
Yidian YeCenter for Immunity and Immunotherapies and the Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA.
Daniel ThomsonCenter for Immunity and Immunotherapies and the Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA.
M Quinn DeGottardiCenter for Immunity and Immunotherapies and the Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA.
Iram F KhanCenter for Immunity and Immunotherapies and the Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA.
Kristina TatiossianGene Therapy Research, CSL Behring, Pasadena, CA 91101, USA.
Brodie A MilesGene Therapy Research, CSL Behring, Pasadena, CA 91101, USA.
Florian AeschimannCSL Behring, Research Bern, 3000 Bern, Switzerland.
Jerome PasquierCSL Behring, Research Bern, 3000 Bern, Switzerland.
Mihee M KimGene Therapy Research, CSL Behring, Pasadena, CA 91101, USA.
David J RawlingsCenter for Immunity and Immunotherapies and the Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Targeted gene editing to restore CD40L expression via homology-directed repair (HDR) in CD34

Indexed as

AAV6AZDCD40LCRISPRDNA-PKcs inhibitorHDRHPSCsT cell

Identifiers

PMID40012884
PMCPMC11863497

What OpenQuestion holds

Textmetadata
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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.