Evidence map›Paper›PMID 42028578›Full record

ArticleMolecular therapy. Nucleic acids2026

Adenine base editor for knockout of proteins: A practical guide from design to analysis with updated MultiEditRbatch.

Ella J Eaton, Bryce J Wick, Jeremy S Chacón, Amy J Wang, Mitchell Kluesner, Jackson T Barnes, Bibekananda Kar, Minjing Wang, Matthew J Johnson, Joseph G Skeate 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. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. 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

12 authors.

Ella J EatonDepartment of Pediatrics, University of Minnesota, Minneapolis, MN, USA.
Bryce J WickDepartment of Pediatrics, University of Minnesota, Minneapolis, MN, USA.
Jeremy S ChacónMinnesota Supercomputing Institute, University of Minnesota, Minneapolis, MN, USA.
Amy J WangDepartment of Pediatrics, University of Minnesota, Minneapolis, MN, USA.
Mitchell KluesnerMolecular and Cellular Biology Graduate Program, University of Washington, Seattle, WA, USA.
Jackson T BarnesDepartment of Immunology, Mayo Clinic, Rochester, MN, USA.
Bibekananda KarDepartment of Pediatrics, University of Minnesota, Minneapolis, MN, USA.
Minjing WangDepartment of Pediatrics, University of Minnesota, Minneapolis, MN, USA.
Matthew J JohnsonDepartment of Pediatrics, University of Minnesota, Minneapolis, MN, USA.
Joseph G SkeateDepartment of Pediatrics, University of Minnesota, Minneapolis, MN, USA.
Beau R WebberDepartment of Pediatrics, University of Minnesota, Minneapolis, MN, USA.
Branden S MoriarityDepartment of Pediatrics, University of Minnesota, Minneapolis, MN, USA.

Funding

In vivo hematopoietic stem cell gene therapy in a novel genetically humanized murine model of SCID-AF31AI181472 · NIAID · UNIVERSITY OF MINNESOTA · PI Ella Joy Eaton · 2025 to 2026
$73k
NIAID NIH HHS F31 AI181472
6 · The paper itself

Abstract

Adenine base editors (ABEs) are a promising yet underutilized tool for inducing protein knockout compared to Cas9 nuclease, owing in part to a lack of user-friendly platforms for reagent design and implementation. Here, we present a comprehensive workflow to achieve high-efficiency gene knockouts with ABE as an alternative to Cas9 nuclease-based approaches. This includes optimized guide RNA (gRNA) design using SpliceR, a web-based application, followed by genomic and functional validation of ABE-mediated knockouts for several target genes. We validated gRNAs for 14 immunologically relevant targets, using both NGG and NG PAM compatible ABE8e-variants in primary immune cells. To facilitate data analysis, we developed MultiEditRbatch, an updated version of MultiEditR as a user-friendly analysis tool with addition of batch mode for high-throughput analysis of Sanger sequencing data. MultiEditRbatch is available as a web-based application and an R package, enabling robust assessment of base editing outcomes.

Indexed as

adenine base editCRISPR-Cas9knockoutMT: RNA/DNA editingprimary immune cellsanger sequencing analysissplice signal disruption

Identifiers

PMID42028578
PMCPMC13101598

What OpenQuestion holds

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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.