Evidence map›Paper›PMID 28624218›Full record

ArticleMolecular therapy. Nucleic acids2017

Targeted Multifunctional Lipid ECO Plasmid DNA Nanoparticles as Efficient Non-viral Gene Therapy for Leber's Congenital Amaurosis.

Da Sun, Bhubanananda Sahu, Songqi Gao, Rebecca M Schur, Amita M Vaidya, Akiko Maeda, Krzysztof Palczewski, Zheng-Rong Lu

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

Article in Molecular therapy. Nucleic acids, 2017. 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
1.8field-weighted citation impact, top 15% 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, 45 citations in OpenAlex.

  1. Article
  2. Review
  3. Review
  4. Review
  5. Review
  6. Review
  7. Review
  8. Article
  9. How Advanced are Nanocarriers for Effective Subretinal Injection?International journal of nanomedicine · 2024
    Review
  10. Successful large gene augmentation of USH2A with non-viral episomal vectors.Molecular therapy : the journal of the American Society of Gene Therapy · 2023
    Article
  11. Article
  12. Review
  13. Review
  14. Effective gene therapy of Stargardt disease with PEG-ECO/Molecular therapy. Nucleic acids · 2022
    Article
  15. Review
  16. Article
  17. Article
  18. In vivo targeted delivery of nucleic acids and CRISPR genome editors enabled by GSH-responsive silica nanoparticles.Journal of controlled release : official journal of the Controlled Release Society · 2021
    Article
  19. Article
  20. 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

8 authors at 1 institution in 1 country.

Da SunCase Center for Biomolecular Engineering and Department of Biomedical Engineering, School of Engineering, Case Western Reserve University, Cleveland, OH 44106, USA.
Bhubanananda SahuDepartment of Ophthalmology and Visual Sciences, School of Medicine, Case Western Reserve University, Cleveland, OH 44106, USA.
Songqi GaoDepartment of Pharmacology and Cleveland Center for Membrane and Structural Biology, School of Medicine, Case Western Reserve University, Cleveland, OH 44140, USA.
Rebecca M SchurCase Center for Biomolecular Engineering and Department of Biomedical Engineering, School of Engineering, Case Western Reserve University, Cleveland, OH 44106, USA.
Amita M VaidyaCase Center for Biomolecular Engineering and Department of Biomedical Engineering, School of Engineering, Case Western Reserve University, Cleveland, OH 44106, USA.
Akiko MaedaDepartment of Ophthalmology and Visual Sciences, School of Medicine, Case Western Reserve University, Cleveland, OH 44106, USA.
Krzysztof PalczewskiDepartment of Pharmacology and Cleveland Center for Membrane and Structural Biology, School of Medicine, Case Western Reserve University, Cleveland, OH 44140, USA.
Zheng-Rong LuCase Center for Biomolecular Engineering and Department of Biomedical Engineering, School of Engineering, Case Western Reserve University, Cleveland, OH 44106, USA. Electronic address: zxl125@case.edu.
Case Western Reserve University · US

Funding

Carotinoids in VisionR01EY020551 · NEI · CASE WESTERN RESERVE UNIVERSITY · PI VON LINTIG, JOHANNES FRIEDRICH · 2011 to 2024
$4.8M
Regulation of Retinal Physiology by micro-RNAsR01EY022326 · NEI · CASE WESTERN RESERVE UNIVERSITY · PI PALCZEWSKI, KRZYSZTOF · 2013 to 2017
$1.8M
NEI NIH HHS R01 EY020551NEI NIH HHS R01 EY022326
6 · The paper itself

Abstract

Development of a gene delivery system with high efficiency and a good safety profile is essential for successful gene therapy. Here we developed a targeted non-viral delivery system using a multifunctional lipid ECO for treating Leber's congenital amaurosis type 2 (LCA2) and tested this in a mouse model. ECO formed stable nanoparticles with plasmid DNA (pDNA) at a low amine to phosphate (N/P) ratio and mediated high gene transfection efficiency in ARPE-19 cells because of their intrinsic properties of pH-sensitive amphiphilic endosomal escape and reductive cytosolic release (PERC). All-trans-retinylamine, which binds to interphotoreceptor retinoid-binding protein (IRBP), was incorporated into the nanoparticles via a polyethylene glycol (PEG) spacer for targeted delivery of pDNA into the retinal pigmented epithelium. The targeted ECO/pDNA nanoparticles provided high GFP expression in the RPE of 1-month-old Rpe65

Indexed as

gene replacement therapyLeber’s congenital amaurosismultifunctional lipidNon-viral gene deliveryRPE65 protein

Identifiers

PMID28624218
PMCPMC5363681
OpenAlexW2593264164

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.