Evidence map›Paper›PMID 23344650›Full record

ArticleMolecular therapy. Nucleic acids2012

A Hyperactive Transposase Promotes Persistent Gene Transfer of a piggyBac DNA Transposon.

Erin R Burnight, Janice M Staber, Pavel Korsakov, Xianghong Li, Benjamin T Brett, Todd E Scheetz, Nancy L Craig, Paul B McCray

Open access · goldAbstract read
In one paragraph

Article in Molecular therapy. Nucleic acids, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 33 papers.

0numbers the graph read from it
0cells of the map it votes in
33citing papers in PubMed
1.6field-weighted citation impact, top 17% 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

33 citing papers in PubMed, 51 citations in OpenAlex.

  1. Article
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  4. Charcot-Marie-Tooth disease type 1E: Clinical Natural History and Molecular Impact ofmedRxiv : the preprint server for health sciences · 2025
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  11. In Situ Programming of CAR T Cells.Annual review of biomedical engineering · 2021
    Review
  12. Review
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  14. Review
  15. In VivoInternational journal of molecular sciences · 2019
    Article
  16. Review
  17. Article
  18. Article
  19. Article
  20. 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

8 authors at 2 institutions in 1 country.

Erin R Burnight1] Interdisciplinary Graduate Program in Genetics, Carver College of Medicine, University of Iowa, Iowa City, Iowa, USA [2] Center for Gene Therapy of Cystic Fibrosis and Other Genetic Diseases, Carver College of Medicine, University of Iowa, Iowa City, Iowa, USA [3] Department of Pediatrics, Carver College of Medicine, University of Iowa, Iowa City, Iowa, USA.
Janice M Staber
Pavel Korsakov
Xianghong Li
Benjamin T Brett
Todd E Scheetz
Nancy L Craig
Paul B McCray
University of Iowa · USJohns Hopkins University · US

Funding

Vector CoreP01HL051670 · NHLBI · UNIVERSITY OF IOWA · PI MCCRAY, PAUL B · 1993 to 2019
$36.5M
Vector Core-Core 2P30DK054759 · NIDDK · UNIVERSITY OF IOWA · PI Alejandro Antonio Pezzulo · 1998 to 2026
$30.5M
Multidisciplinary Lung Research Career Development ProgramT32HL007638 · NHLBI · UNIVERSITY OF IOWA · PI WELSH, MICHAEL J., ZABNER, JOSEPH · 1986 to 2020
$16.9M
Molecular and Cellular Research to Advance Childhood HealthK12HD027748 · NICHD · UNIVERSITY OF IOWA · PI ALEXANDER G BASSUK · 2002 to 2026
$9.2M
PREDOCTORAL TRAINING PROGRAM IN GENETICST32GM008629 · NIGMS · UNIVERSITY OF IOWA · PI EBERL, DANIEL F · 1997 to 2021
$4.1M
FIV Vectors for the Treatment of Hemophilia AR44HL081976 · NHLBI · VIROGENICS, INC. · PI RASCHKE, WILLIAM C · 2010 to 2012
$2.7M
Bioinformatics Training in the Life Sciences and BiomedicineT32GM082729 · NIGMS · UNIVERSITY OF IOWA · PI CASAVANT, THOMAS L. · 2009 to 2013
$544k
NHLBI NIH HHS P01 HL051670NHLBI NIH HHS R44 HL081976NHLBI NIH HHS T32 HL007638NICHD NIH HHS K12 HD027748NIDDK NIH HHS P30 DK054759NIGMS NIH HHS T32 GM008629NIGMS NIH HHS T32 GM082729
6 · The paper itself

Abstract

Nonviral vector systems are used increasingly in gene targeting and gene transfer applications. The piggyBac transposon represents an alternative integrating vector for in vivo gene transfer. We hypothesized that this system could achieve persistent gene transfer to the liver when administered systemically. We report that a novel hyperactive transposase generated higher transposition efficiency than a codon-optimized transposase in a human liver cell line. Hyperactive transposase-mediated reporter gene expression persisted at levels twice that of codon-optimized transposase in the livers of mice for the 6-month study. Of note, expression persisted in mice following partial hepatectomy, consistent with expression from an integrated transgene. We also used the hyperactive transposase to deliver the human α(1)-antitrypsin gene and achieved stable expression in serum. To determine the integration pattern of insertions, we performed large-scale mapping in human cells and recovered 60,685 unique hyperactive transposase-mediated insertions. We found that a hyperactive piggyBac transposase conferred an altered pattern of integration from that of insect piggyBac transposase, with a decreased frequency of integration near transcription start sites than previously reported. Our results support that the piggyBac transposon combined with the hyperactive transposase is an efficient integrating vector system for in vitro and in vivo applications.Molecular Therapy - Nucleic Acids (2012) 1, e50; doi:10.1038/mtna.2012.12; published online 16 October 2012.

Identifiers

PMID23344650
PMCPMC3499692
OpenAlexW2028349812

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.