Evidence map›Paper›PMID 28530135›Full record

ArticleHuman gene therapy2017

Prolonged Expression of Secreted Enzymes in Dogs After Liver-Directed Delivery of Sleeping Beauty Transposons: Implications for Non-Viral Gene Therapy of Systemic Disease.

Elena L Aronovich, Kendra A Hyland, Bryan C Hall, Jason B Bell, Erik R Olson, Myra Urness Rusten, David W Hunter, N Matthew Ellinwood, R Scott McIvor, Perry B Hackett

Open access · hybridAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Human gene therapy, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.

0numbers the graph read from it
0cells of the map it votes in
7citing papers in PubMed
0.7field-weighted citation impact, top 24% 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

7 citing papers in PubMed, 9 citations in OpenAlex.

  1. A Preliminary Evaluation ofPharmaceutics · 2026
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4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

10 authors at 3 institutions in 1 country.

Elena L Aronovich1 Department of Genetics, Cell Biology and Development and Center for Genome Engineering, University of Minnesota , Minneapolis, Minnesota.
Kendra A Hyland2 Discovery Genomics, Inc. , Minneapolis, Minnesota.
Bryan C Hall1 Department of Genetics, Cell Biology and Development and Center for Genome Engineering, University of Minnesota , Minneapolis, Minnesota.
Jason B Bell1 Department of Genetics, Cell Biology and Development and Center for Genome Engineering, University of Minnesota , Minneapolis, Minnesota.
Erik R Olson2 Discovery Genomics, Inc. , Minneapolis, Minnesota.
Myra Urness Rusten3 Department of Radiology, University of Minnesota , Minneapolis, Minnesota.
David W Hunter3 Department of Radiology, University of Minnesota , Minneapolis, Minnesota.
N Matthew Ellinwood4 Department of Animal Science, Iowa State University , Ames, Iowa.
R Scott McIvor1 Department of Genetics, Cell Biology and Development and Center for Genome Engineering, University of Minnesota , Minneapolis, Minnesota.
Perry B Hackett1 Department of Genetics, Cell Biology and Development and Center for Genome Engineering, University of Minnesota , Minneapolis, Minnesota.
University of Minnesota · USID Genomics (United States) · USIowa State University · US

Funding

THERAPY FOR HYPERAMMONEMIA WITH GENETICALLY ENGINEERED BACTERIAP01HD032652 · NICHD · UNIVERSITY OF MINNESOTA TWIN CITIES · PI WHITLEY, CHESTER B. · 1995 to 2016
$14.5M
Sleeping Beauty-Mediated Gene Therapy for HemophiliaR44HL072539 · NHLBI · DISCOVERY GENOMICS, INC. · PI MCIVOR, R. SCOTT · 2005 to 2013
$3.0M
SB Transposons for Gene TherapyR01DK082516 · NIDDK · UNIVERSITY OF MINNESOTA · PI HACKETT, PERRY B. · 2010 to 2013
$1.3M
Delivery of Sleeping Beauty Transposons to Dog Liver for Gene TherapyR41DK081249 · NIDDK · DISCOVERY GENOMICS, INC. · PI HACKETT, PERRY B. · 2008 to 2008
$291k
NHLBI NIH HHS R44 HL072539NICHD NIH HHS P01 HD032652NIDDK NIH HHS R01 DK082516NIDDK NIH HHS R41 DK081249
6 · The paper itself

Abstract

The non-viral, integrating Sleeping Beauty (SB) transposon system is efficient in treating systemic monogenic disease in mice, including hemophilia A and B caused by deficiency of blood clotting factors and mucopolysaccharidosis types I and VII caused by α-L-iduronidase (IDUA) and β-glucuronidase (GUSB) deficiency, respectively. Modified approaches of the hydrodynamics-based procedure to deliver transposons to the liver in dogs were recently reported. Using the transgenic canine reporter secreted alkaline phosphatase (cSEAP), transgenic protein in the plasma was demonstrated for up to 6 weeks post infusion. This study reports that immunosuppression of dogs with gadolinium chloride (GdCl

Indexed as

Genetic TherapyGene Transfer TechniquesAnimalsDNA Transposable ElementsDogsGadoliniumGene ExpressionGenes, ReporterGlucuronidaseHemophilia AIduronidaseImmunomodulationLiverMaleMice, Inbred C57BLTransgenesDNA Transposable ElementsGadoliniumGlucuronidaseIduronidaseTransposaseshemophiliahydrodynamics-based deliveryimmune responsemonogenic diseasemucopolysaccharidosis

Identifiers

PMID28530135
PMCPMC5549796
OpenAlexW2615955058

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.