Evidence map›Paper›PMID 24399204›Full record

ArticleMolecular therapy. Nucleic acids2014

Micro-minicircle Gene Therapy: Implications of Size on Fermentation, Complexation, Shearing Resistance, and Expression.

Sofia Stenler, Oscar Pb Wiklander, Maria Badal-Tejedor, Janne Turunen, Joel Z Nordin, David Hallengärd, Britta Wahren, Samir El Andaloussi, Mark W Rutland, C I Edvard Smith and 2 more

Open access · goldAbstract read
In one paragraph

Article in Molecular therapy. Nucleic acids, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers.

0numbers the graph read from it
0cells of the map it votes in
21citing papers in PubMed
2.4field-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

21 citing papers in PubMed, 34 citations in OpenAlex.

  1. Article
  2. Review
  3. Article
  4. Review
  5. Article
  6. Article
  7. Article
  8. A Novel Cre Recombinase-MediatedApplied and environmental microbiology · 2019
    Article
  9. Article
  10. Nonviral Gene Therapy for Cancer: A Review.Diseases (Basel, Switzerland) · 2018
    Review
  11. Article
  12. Review
  13. Article
  14. Article
  15. Article
  16. Article
  17. Article
  18. Article
  19. Detecting cancers through tumor-activatable minicircles that lead to a detectable blood biomarker.Proceedings of the National Academy of Sciences of the United States of America · 2015
    Article
  20. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

12 authors at 4 institutions in 2 countries.

Sofia StenlerDepartment of Laboratory Medicine, Clinical Research Center, Karolinska Institutet, Stockholm, Sweden.
Oscar Pb WiklanderDepartment of Laboratory Medicine, Clinical Research Center, Karolinska Institutet, Stockholm, Sweden.
Maria Badal-TejedorYKI, Institute for Surface Chemistry, Stockholm, Sweden.
Janne TurunenDepartment of Laboratory Medicine, Clinical Research Center, Karolinska Institutet, Stockholm, Sweden.
Joel Z NordinDepartment of Laboratory Medicine, Clinical Research Center, Karolinska Institutet, Stockholm, Sweden.
David HallengärdDepartment of Microbiology, Tumor and Cell Biology, Karolinska Institutet, Stockholm, Sweden.
Britta WahrenDepartment of Microbiology, Tumor and Cell Biology, Karolinska Institutet, Stockholm, Sweden.
Samir El Andaloussi1] Department of Laboratory Medicine, Clinical Research Center, Karolinska Institutet, Stockholm, Sweden [2] Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK.
Mark W RutlandDepartment of Chemistry, Surface Chemistry, Royal Institute of Technology, Stockholm, Sweden.
C I Edvard SmithDepartment of Laboratory Medicine, Clinical Research Center, Karolinska Institutet, Stockholm, Sweden.
Karin E LundinDepartment of Laboratory Medicine, Clinical Research Center, Karolinska Institutet, Stockholm, Sweden.
Pontus Blomberg1] Department of Laboratory Medicine, Clinical Research Center, Karolinska Institutet, Stockholm, Sweden [2] Vecura, Clinical Research Center, Karolinska University Hospital, Stockholm, Sweden.
Karolinska Institutet · SEKarolinska University Hospital · SEKTH Royal Institute of Technology · SEUniversity of Oxford · GB

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The minicircle (MC), composed of eukaryotic sequences only, is an interesting approach to increase the safety and efficiency of plasmid-based vectors for gene therapy. In this paper, we investigate micro-MC (miMC) vectors encoding small regulatory RNA. We use a construct encoding a splice-correcting U7 small nuclear RNA, which results in a vector of 650 base pairs (bp), as compared to a conventional 3600 bp plasmid carrying the same expression cassette. Furthermore, we construct miMCs of varying sizes carrying different number of these cassettes. This allows us to evaluate how size influences production, super-coiling, stability and efficiency of the vector. We characterize coiling morphology by atomic force microscopy and measure the resistance to shearing forces caused by an injector device, the Biojector. We compare the behavior of miMCs and plasmids in vitro using lipofection and electroporation, as well as in vivo in mice. We here show that when the size of the miMC is reduced, the formation of dimers and trimers increases. There seems to be a lower size limit for efficient expression. We demonstrate that miMCs are more robust than plasmids when exposed to shearing forces, and that they show extended expression in vivo.Molecular Therapy-Nucleic Acids (2014); doi:10.1038/mtna.2013.67.

Identifiers

PMID24399204
PMCPMC3910003
OpenAlexW1975651709

What OpenQuestion holds

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Registered trials

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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.