Evidence map›Paper›PMID 18653181›Full record

ArticleExperimental eye research2008

Non-invasive gene transfer by iontophoresis for therapy of an inherited retinal degeneration.

Eric H Souied, Silvia N M Reid, Natik I Piri, Leonid E Lerner, Steven Nusinowitz, Debora B Farber

Abstract readEvaluation Study
In one paragraph

Article in Experimental eye research, 2008. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.

0numbers the graph read from it
0cells of the map it votes in
16citing papers in PubMed
1.2field-weighted citation impact, top 22% 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

16 citing papers in PubMed, 39 citations in OpenAlex.

  1. Review
  2. Review
  3. Review
  4. Minicircle Delivery to the Neural Retina as a Gene Therapy Approach.International journal of molecular sciences · 2022
    Review
  5. Review
  6. Review
  7. Article
  8. Review
  9. Trans-ocular Electric CurrentMolecular therapy. Methods & clinical development · 2019
    Article
  10. Article
  11. Article
  12. Gene Therapy of ABCA4-Associated Diseases.Cold Spring Harbor perspectives in medicine · 2015
    Review
  13. Vector platforms for gene therapy of inherited retinopathies.Progress in retinal and eye research · 2014
    Review
  14. Article
  15. Review
  16. Review
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

6 authors at 1 institution in 1 country.

Eric H SouiedJules Stein Eye Institute, UCLA School of Medicine, Los Angeles, CA 90095, USA.
Silvia N M Reid
Natik I Piri
Leonid E Lerner
Steven Nusinowitz
Debora B Farber
Doheny Eye Institute · US

Funding

PHOSPHODIESTERASES IN PHOTORECEPTOR METABOLISM &DISEASER01EY002651 · NEI · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI FARBER, DEBORA B · 1985 to 2003
$1.9M
NEI NIH HHS R01 EY002651NEI NIH HHS R01EY02651
6 · The paper itself

Abstract

Despite extensive research on many of the genes responsible for inherited retinal degenerations leading to blindness, no effective treatment is currently available for patients affected with these diseases. Among the therapeutic approaches tested on animal models of human retinal degeneration, gene therapy using different types of viral vectors as delivery agents has yielded promising results. We report here our results on a non-invasive, non-viral delivery approach using transscleral iontophoresis for transfer of plasmid DNA into mouse retina. Proof of principle experiments were carried out using plasmid containing GFP cDNA to demonstrate expression of the transferred gene in the retina after single applications of iontophoresis. Various parameters for multiple applications of iontophoresis were optimized to sustain GFP gene expression in mouse photoreceptors. Subsequently, repeated iontophoresis of plasmid containing normal cGMP-phosphodiesterase beta-subunit (beta-PDE) cDNA was performed in the rd1 mouse, an animal model of autosomal recessive retinitis pigmentosa caused by a mutant beta-PDE gene. In normal mice, transscleral iontophoresis of the GFP plasmid provided a significant increase in fluorescence of the retina in the treated versus non-treated eyes. In rd1 mice, repeated iontophoresis of beta-PDE cDNA plasmid partially rescued photoreceptors morphologically, as observed by microscopy, and functionally, as recorded on ERG measurements, without adverse effects. Therefore, transscleral iontophoresis of plasmid DNA containing therapeutic genes may be an efficient, safe and non-invasive method for the treatment of retinal degenerations.

Indexed as

Gene Transfer TechniquesAnimalsBlotting, WesternCyclic Nucleotide Phosphodiesterases, Type 6Disease Models, AnimalDNA, ComplementaryElectroretinographyGenetic TherapyGreen Fluorescent ProteinsIontophoresisMiceMice, Inbred C57BLMice, Mutant StrainsMicroscopy, ConfocalPlasmidsRetinitis PigmentosaCyclic Nucleotide Phosphodiesterases, Type 6DNA, ComplementaryGreen Fluorescent ProteinsPde6b protein, mouse

Identifiers

PMID18653181
PMCPMC2713253
OpenAlexW2059101360

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.