Evidence map›Paper›PMID 38094200›Full record

ArticleMolecular therapy. Methods & clinical development2023

Analysis of vector genome integrations in multicentric lymphoma after AAV gene therapy in a severe hemophilia A dog.

Lucas Van Gorder, Bhavya S Doshi, Elinor Willis, Timothy C Nichols, Emma Cook, John K Everett, Elizabeth P Merricks, Valder R Arruda, Frederic D Bushman, Mary Beth Callan and 1 more

Open access · goldAbstract read
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
2.2field-weighted citation impact, top 11% of its field
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

4 citing papers in PubMed, 7 citations in OpenAlex.

  1. Article
  2. Malignancy and gene therapy in hemophilia.Research and practice in thrombosis and haemostasis · 2026
    Article
  3. Pilocytic astrocytoma in a child with spinal muscular atrophy treated with onasemnogene abeparvovec.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Article
  4. 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

11 authors at 2 institutions in 1 country.

Lucas Van GorderDepartment of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Bhavya S DoshiDepartment of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Elinor WillisDepartment of Pathobiology, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Timothy C NicholsDepartment of Pathology and Laboratory Medicine and the UNC Blood Research Center, University of North Carolina, Chapel Hill, NC 27599, USA.
Emma CookDepartment of Microbiology Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
John K EverettDepartment of Microbiology Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Elizabeth P MerricksDepartment of Pathology and Laboratory Medicine and the UNC Blood Research Center, University of North Carolina, Chapel Hill, NC 27599, USA.
Valder R ArrudaDepartment of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Frederic D BushmanDepartment of Microbiology Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Mary Beth CallanDepartment of Clinical Sciences and Advanced Medicine, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Benjamin J Samelson-JonesDepartment of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
University of Pennsylvania · USChildren's Hospital of Philadelphia · US

Funding

UNIVERSITY OF PENNSYLVANIA CAN CTR SUPPORT GRANTP30CA016520 · NCI · UNIVERSITY OF PENNSYLVANIA · PI SHELLEY L BERGER · 1985 to 2026
$222.3M
Virus & Reservoirs CoreP30AI045008 · NIAID · UNIVERSITY OF PENNSYLVANIA · PI ROBERT GROSS · 1999 to 2026
$78.6M
BEAT-HIV: Delaney Collaboratory to Cure HIV-1 Infection by Combination ImmunotherapyUM1AI164570 · NIAID · WISTAR INSTITUTE · PI Luis J Montaner, James L. Riley · 2021 to 2026
$34.7M
HEMATOLOGY CLINICAL RESEARCH TRAINING PROGRAMT32HL007439 · NHLBI · UNIVERSITY OF PENNSYLVANIA · PI LAWRENCE F BRASS, PETER S KLEIN · 1985 to 2026
$17.1M
Modeling HIV CAR-T cell trafficking and persistence in Non-Human PrimatesU19AI149680 · NIAID · UNIVERSITY OF PENNSYLVANIA · PI BUSHMAN, FREDERIC D · 2020 to 2024
$13.2M
Gene Therapy for SCID-X1 with Low Dose Busulfan and a SIN-lentiviral VectorU01AI125051 · NIAID · BOSTON CHILDREN'S HOSPITAL · PI DAVID A WILLIAMS · 2016 to 2026
$9.7M
A Phase I Clinical Trial Testing Feasibility of Hematopoietic Stem Cell Gene Therapy Using Platelet Factor VIII to Safely Improve Hemostasis for Severe Hemophilia A with Inhibitory AntibodiesR01HL142791 · NHLBI · MEDICAL COLLEGE OF WISCONSIN · PI EAPEN, MARY, WILCOX, DAVID ALLEN · 2019 to 2023
$8.2M
Project 3: Defining adaptive immune interactions that shape Clostridioides difficile infectionU19AI174998 · NIAID · UNIVERSITY OF PENNSYLVANIA · PI Michael C. Abt · 2023 to 2026
$7.6M
Linking insertional mutagenesis and cell function to improve CAR T cell therapyR01CA241762 · NCI · UNIVERSITY OF PENNSYLVANIA · PI BUSHMAN, FREDERIC D, MELENHORST, JAN J · 2019 to 2023
$3.1M
Immune tolerance induction by AAV-FVIII gene therapy for canine hemophilia A with inhibitorsR01HL158781 · NHLBI · CHILDREN'S HOSP OF PHILADELPHIA · PI SAMELSON-JONES, BEN J · 2021 to 2024
$3.0M
Preserving Genome Integrity In AAV-Mediated Gene TherapyR01HL160748 · NHLBI · CHILDREN'S HOSP OF PHILADELPHIA · PI BUSHMAN, FREDERIC D, SABATINO, DENISE E · 2022 to 2025
$2.6M
Mechanistic insights into factor VIII inhibitor formation and eradicationK99HL156073 · NHLBI · EMORY UNIVERSITY · PI DOSHI, BHAVYA SHARAD · 2021 to 2025
$786k
NCI NIH HHS P30 CA016520NCI NIH HHS R01 CA241762NHLBI NIH HHS K99 HL156073NHLBI NIH HHS R01 HL142791NHLBI NIH HHS R01 HL158781NHLBI NIH HHS R01 HL160748NHLBI NIH HHS T32 HL007439NIAID NIH HHS P30 AI045008NIAID NIH HHS U01 AI125051NIAID NIH HHS U19 AI149680NIAID NIH HHS U19 AI174998NIAID NIH HHS UM1 AI164570
6 · The paper itself

Abstract

Adeno-associated viral (AAV) vectors have traditionally been viewed as predominantly nonintegrating, with limited concerns for oncogenesis. However, accumulating preclinical data have shown that AAV vectors integrate more often than previously appreciated, with the potential for genotoxicity. To understand the consequences of AAV vector integration, vigilance for rare genotoxic events after vector administration is essential. Here, we investigate the development of multicentric lymphoma in a privately owned dog, PC9, with severe hemophilia A that was treated with an AAV8 vector encapsidating a B domain-deleted canine coagulation

Indexed as

AAVcancerdog modelgene therapygenotoxicityintegrationlymphoma

Identifiers

PMID38094200
PMCPMC10716008
OpenAlexW4388673135

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.