Evidence map›Paper›PMID 11526457›Full record

ArticleGene therapy2001

Electrotransfer of naked DNA in the skeletal muscles of animal models of muscular dystrophies.

J T Vilquin, P F Kennel, M Paturneau-Jouas, P Chapdelaine, N Boissel, P Delaère, J P Tremblay, D Scherman, M Y Fiszman, K Schwartz

Abstract readComparative Study
PubMed Publisher
In one paragraph

Article in Gene therapy, 2001. 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
5.7field-weighted citation impact, top 3% 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, 99 citations in OpenAlex.

  1. Article
  2. Article
  3. CORP: Gene delivery into murine skeletal muscle using in vivo electroporation.Journal of applied physiology (Bethesda, Md. : 1985) · 2022
    Review
  4. Article
  5. Review
  6. Article
  7. Article
  8. Article
  9. Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.Molecular therapy : the journal of the American Society of Gene Therapy · 2011
    Review
  10. Gene therapy for muscular dystrophies: progress and challenges.Journal of clinical neurology (Seoul, Korea) · 2010
    Article
  11. Article
  12. Ca(2+) sparks operated by membrane depolarization require isoform 3 ryanodine receptor channels in skeletal muscle.Proceedings of the National Academy of Sciences of the United States of America · 2007
    Article
  13. Review
  14. Article
  15. Article
  16. Review
  17. Article
  18. Article
  19. Review
  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

10 authors at 3 institutions in 2 countries.

J T VilquinINSERM U 523, Hôpital de la Salpêtrière, Paris, France.
P F Kennel
M Paturneau-Jouas
P Chapdelaine
N Boissel
P Delaère
J P Tremblay
D Scherman
M Y Fiszman
K Schwartz
Inserm · FRCentre hospitalier de l'Université Laval · CACentre National de la Recherche Scientifique · FR

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The electrotransfer of naked DNA has recently been adapted to the transduction of skeletal muscle fibers. We investigated the short- and long-term efficacy of this methodology in wild-type animals and in mouse models of congenital muscular dystrophy (dy/dy, dy(2J)/dy(2J)), or Duchenne muscular dystrophy (mdx/mdx). Using a reporter construct, the short-term efficacy of fiber transduction reached 40% and was similar in wild-type, dy/dy and dy(2J)/dy(2J) animals, indicating that ongoing muscle fibrosis was not a major obstacle to the electrotransfer-mediated gene transfer. Although the complete rejection of transduced fibers was observed within 3 weeks in the absence of immunosuppression, the persistency was prolonged over 10 weeks when transient or continuous immunosuppressive regimens were used. Using therapeutic plasmids, we demonstrated that electrotransfer also allowed the transduction of large constructs encoding the laminin alpha2 chain in dy/dy mouse, or a chimeric dystrophin-EGFP protein in mdx/mdx mouse. The correct sarcolemmal localization of these structural proteins demonstrated the functional relevance of their expression in vivo, with a diffusion domain estimated to be 300 to 500 microm. However, degeneration-regeneration events hampered the long-term stability of transduced fibers. Given its efficacy for naked DNA transfer in these models of muscular dystrophies, and despite some limitations, gene electrotransfer methodology should be further explored as a potential avenue for treatment of muscular dystrophies.

Indexed as

AnimalsDystrophinElectroporationGene ExpressionGenetic TherapyGreen Fluorescent ProteinsHindlimbLamininLuminescent ProteinsMiceMice, Inbred mdxModels, AnimalMuscle, SkeletalMuscular DystrophiesMuscular Dystrophy, DuchennePlasmidsDystrophinGreen Fluorescent ProteinsLamininLuminescent Proteins

Identifiers

PMID11526457
OpenAlexW2326611690

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.