Evidence map›Paper›PMID 41286106›Full record

ReviewNature genetics2026

Measurement and clinical interpretation of CRISPR off-targets.

Ariella Angelini Stewart, Rebecca C Ahrens-Nicklas, Shengdar Q Tsai, Kiran Musunuru, Petros Giannikopoulos, Claire D Clelland

Abstract readReview
In one paragraph

Review in Nature genetics, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing 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

6 citing papers in PubMed.

  1. Review
  2. Review
  3. Article
  4. Considerations for early life genetic therapies in cystic fibrosis.American journal of physiology. Lung cellular and molecular physiology · 2026
    Review
  5. Review
  6. 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.

Ariella Angelini StewartWeill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.ORCID 0009-0004-4819-5609
Rebecca C Ahrens-NicklasThe Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Shengdar Q TsaiSt. Jude Children's Research Hospital, Memphis, TN, USA.ORCID 0000-0001-9161-3993
Kiran MusunuruThe Children's Hospital of Philadelphia, Philadelphia, PA, USA.ORCID 0000-0003-3298-0368
Petros GiannikopoulosInnovative Genomics Institute, Berkeley, CA, USA.ORCID 0000-0003-4342-1756
Claire D ClellandWeill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA. claire.clelland@ucsf.edu.ORCID 0000-0002-9112-2278

Funding

Resource Core II: In Vivo CoreU19NS132303 · NINDS · UNIVERSITY OF CALIFORNIA BERKELEY · PI NIREN MURTHY · 2023 to 2026
$19.7M
CRISPR-Cas9 editing in C9orf72 patient derived iPSCK08NS112330 · NINDS · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI CLELLAND, CLAIRE · 2020 to 2024
$1.2M
Optimization of CRISPR genome editor and its delivery strategy for C9orf72 frontotemporal dementiaU01NS134062 · NINDS · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI CLELLAND, CLAIRE, GONG, SHAOQIN - · 2023 to 2023
$870k
NINDS NIH HHS K08 NS112330NINDS NIH HHS U01 NS134062NINDS NIH HHS U19 NS132303
6 · The paper itself

Abstract

CRISPR genetic therapies are revolutionizing the landscape of preclinical research and clinical studies, providing new potential routes for curative intervention for a range of previously untreatable diseases. As with any therapy, the therapeutic benefits and risks must be weighed against consideration of the disease threat. Genome-related adverse events are an inherent risk of CRISPR genetic therapies, including off-target edits. The perception that CRISPR therapies ought to have near-zero off-targets belies clinical medicine, therapy development and biology, which demonstrate that 'perfect' therapeutics do not exist. Given that not all genomic off-target events are equal, we provide a practical framework to evaluate and assess off-target safety based on the tools available today and ones that will be developed in the future. With the comprehensive information and assessment gathered using these guidelines, we aim to streamline the transition of CRISPR therapeutics from bench to bedside.

Indexed as

Clustered Regularly Interspaced Short Palindromic RepeatsCRISPR-Cas SystemsGene EditingGenetic TherapyAnimalsHumans

Identifiers

PMID41286106
PMCPMC12927645

What Socratic holds

Textmetadata
LicenceTDM
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.