Evidence map›Paper›PMID 42306087›Full record

ArticleMolecular therapy. Nucleic acids2026

Comprehensive assessment of on- and off-target mutagenesis via lipid nanoparticle delivery of CRISPR-Cas9 genome editing.

Youichi Naoe, Naoko Fujimoto, Yukimasa Makita, Dongyang Li, Naoto Inukai, Akitsu Hotta

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. Review
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

6 authors.

Youichi NaoeDepartment of Clinical Application, Center for IPS Cell Research and Application (CiRA), Kyoto University, Kyoto, Japan.
Naoko FujimotoDepartment of Clinical Application, Center for IPS Cell Research and Application (CiRA), Kyoto University, Kyoto, Japan.
Yukimasa MakitaTakeda-CiRA Joint Program for IPS Cell Applications (T-CiRA), Fujisawa, Kanagawa, Japan.
Dongyang LiDepartment of Clinical Application, Center for IPS Cell Research and Application (CiRA), Kyoto University, Kyoto, Japan.
Naoto InukaiTakeda-CiRA Joint Program for IPS Cell Applications (T-CiRA), Fujisawa, Kanagawa, Japan.
Akitsu HottaDepartment of Clinical Application, Center for IPS Cell Research and Application (CiRA), Kyoto University, Kyoto, Japan.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Ensuring the safety of CRISPR-Cas9 genome editing requires comprehensive assessment of off-target mutagenesis, particularly for therapeutic applications under regulatory review. DNA-free lipid nanoparticle (LNP) delivery is expected to minimize insertional risks compared to adeno-associated virus (AAV) vectors, but experimental validation has been limited. Here, on-target amplicon sequencing in mouse muscle demonstrated insertions largely derived from host genomic sequences, with no detectable AAV or transgene integration. Benchmarking 13

Indexed as

CRISPR-Cas9DMDdual gRNAsDuchenne muscular dystrophyexon skippingindel clusterlipid nanoparticleLNPMT: RNA/DNA editingoff-target riskWGSwhole-genome sequencing

Identifiers

PMID42306087
PMCPMC13267559

What Socratic 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 Socratic graph.