Evidence map›Paper›PMID 42603971›Full record

ArticleMolecular therapy. Advances2026

In-depth characterization of stem cell potency and genotoxicity for clinical-scale

Asma Naseem, Winston Vetharoy, Thomas E Whittaker, Federica Zinghirino, Antonio Carusillo, Roopkatha Ghosh, Eduardo Gomez-Castaneda, Hashim Ali, Carlo Cipriani, Eugenio Montini and 5 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Advances, 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

15 authors.

Asma NaseemInfection, Immunity & Inflammation Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, University College London, 20 Guilford Street, London WC1N 1DZ, UK.
Winston VetharoyInfection, Immunity & Inflammation Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, University College London, 20 Guilford Street, London WC1N 1DZ, UK.
Thomas E WhittakerInfection, Immunity & Inflammation Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, University College London, 20 Guilford Street, London WC1N 1DZ, UK.
Federica ZinghirinoInfection, Immunity & Inflammation Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, University College London, 20 Guilford Street, London WC1N 1DZ, UK.
Antonio CarusilloCenter for Genomic Medicine and Rare Diseases, Department of Medical and Surgical Sciences for Children and Adults, University of Modena and Reggio Emilia, Modena, Italy.
Roopkatha GhoshInfection, Immunity & Inflammation Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, University College London, 20 Guilford Street, London WC1N 1DZ, UK.
Eduardo Gomez-CastanedaInfection, Immunity & Inflammation Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, University College London, 20 Guilford Street, London WC1N 1DZ, UK.
Hashim AliDivision of Infection and Immunity, University College London, London, UK.
Carlo CiprianiSan Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Hospital, Milan, Italy.
Eugenio MontiniSan Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Hospital, Milan, Italy.
Adrian J ThrasherInfection, Immunity & Inflammation Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, University College London, 20 Guilford Street, London WC1N 1DZ, UK.
Giorgia SantilliInfection, Immunity & Inflammation Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, University College London, 20 Guilford Street, London WC1N 1DZ, UK.
Daniela CesanaSan Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Hospital, Milan, Italy.
Giandomenico TurchianoInfection, Immunity & Inflammation Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, University College London, 20 Guilford Street, London WC1N 1DZ, UK.
Alessia CavazzaInfection, Immunity & Inflammation Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, University College London, 20 Guilford Street, London WC1N 1DZ, UK.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Translating CRISPR-Cas9-based homology-directed repair (HDR) strategies into clinical application remains a major challenge due to limited standardization, concerns over safety, and efficacy issues. Here, we present a comprehensive and clinically compliant preclinical framework for the

Indexed as

gene editinghematopoietic stem and progenitor cellshomology-directed repairpreclinical

Identifiers

PMID42603971
PMCPMC13476499

What OpenQuestion holds

Textmetadata
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 OpenQuestion graph.