Evidence map›Paper›PMID 40450520›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2025

Quantifying the mutational landscape of retroviral and lentiviral vectors in gene therapy patients.

Kevyn L Hart, Ralph Valentine Crisostomo, Annika Mittelhauser, Lingyu Zhan, Nika Kononov, Kathryn Bradford, Donald B Kohn

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Article
  2. Review
  3. APOBEC3 and the mutational signature of lentiviral vectors.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    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

7 authors.

Kevyn L HartDepartment of Human Genetics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA 90095, USA.
Ralph Valentine CrisostomoMolecular Biology Interdepartmental Program, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA 90095, USA.
Annika MittelhauserDepartment of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA.
Lingyu ZhanDepartment of Psychiatry, Center for Neurobehavioral Genetics, Semel Institute for Neuroscience and Human Behavior, University of California, Los Angeles, Los Angeles, CA 90095, USA; The Collaboratory, Institute for Quantitative and Computational Biosciences, University of California, Los Angeles, Los Angeles, CA 90095, USA.
Nika KononovThe Collaboratory, Institute for Quantitative and Computational Biosciences, University of California, Los Angeles, Los Angeles, CA 90095, USA.
Kathryn BradfordDepartment of Pediatrics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA.
Donald B KohnDepartment of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA; Department of Pediatrics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA; Department of Molecular and Medical Pharmacology, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA; The Eli & Edythe Broad Center of Regenerative Medicine & Stem Cell Research, University of California, Los Angeles, Los Angeles, CA 90095, USA. Electronic address: e-dkohn1@mednet.ucla.edu.

Funding

UCLA Clinical Translational Science InstituteUL1TR001881 · NCATS · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI ARLEEN F. BROWN, ARASH NAEIM · 2016 to 2026
$118.1M
Training CoreTL1DK132768 · NIDDK · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI Elizabeta Nemeth · 2021 to 2026
$3.4M
NCATS NIH HHS UL1 TR001881NIDDK NIH HHS TL1 DK132768
6 · The paper itself

Abstract

Adenosine deaminase severe combined immunodeficiency (ADA-SCID) is a monogenic disorder caused by mutations in the ADA gene. Gene therapy using γ-retroviral and lentiviral vector gene addition approaches have shown curative results. We sequenced the ADA transgene in transduced CD3

Indexed as

Adenosine DeaminaseAgammaglobulinemiaGenetic TherapyGenetic VectorsLentivirusMutationRetroviridaeSevere Combined ImmunodeficiencyAPOBEC DeaminasesHEK293 CellsHumansT-LymphocytesTransduction, GeneticTransgenesAdenosine DeaminaseAPOBEC3 proteins, humanAPOBEC DeaminasesADA-SCIDAPOBEC3gene therapylentiviral vectorsreverse transcriptase

Identifiers

PMID40450520
PMCPMC12432904

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.