Evidence map›Paper›PMID 23837668›Full record

ArticleACS nano2013

Development of poly(β-amino ester)-based biodegradable nanoparticles for nonviral delivery of minicircle DNA.

Michael Keeney, Sang-Ging Ong, Amanda Padilla, Zhenyu Yao, Stuart Goodman, Joseph C Wu, Fan Yang

Open access · greenAbstract read
In one paragraph

Article in ACS nano, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 34 papers.

0numbers the graph read from it
0cells of the map it votes in
34citing papers in PubMed
5.4field-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

34 citing papers in PubMed, 85 citations in OpenAlex.

  1. Article
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  12. Article
  13. Biodegradable Polymers for Gene Delivery.Molecules (Basel, Switzerland) · 2019
    Review
  14. Article
  15. Article
  16. Article
  17. DNA Vaccines-How Far From Clinical Use?International journal of molecular sciences · 2018
    Review
  18. Article
  19. Article
  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

7 authors at 2 institutions in 1 country.

Michael KeeneyDepartment of Orthopedic Surgery, Stanford University, Stanford, California 94305, United States.
Sang-Ging Ong
Amanda Padilla
Zhenyu Yao
Stuart Goodman
Joseph C Wu
Fan Yang
Stanford University · USCalifornia Institute for Regenerative Medicine · US

Funding

Molecular Imaging of Targeted Cardiac Gene TherapyR01HL093172 · NHLBI · STANFORD UNIVERSITY · PI WU, JOSEPH C. · 2009 to 2015
$2.7M
Integrated Strategies for Novel Treatment of Myocardial IschemiaR01HL095571 · NHLBI · STANFORD UNIVERSITY · PI WU, JOSEPH C. · 2010 to 2014
$2.0M
NHLBI NIH HHS R01 HL093172NHLBI NIH HHS R01 HL095571
6 · The paper itself

Abstract

Gene therapy provides a powerful tool for regulating cellular processes and tissue repair. Minicircle (MC) DNA are supercoiled DNA molecules free of bacterial plasmid backbone elements and have been reported to enhance prolonged gene expression compared to conventional plasmids. Despite the great promise of MC DNA for gene therapy, methods for safe and efficient MC DNA delivery remain lacking. To overcome this bottleneck, here we report the development of a poly(β-amino ester) (PBAE)-based, biodegradable nanoparticulate platform for efficient delivery of MC DNA driven by a Ubc promoter in vitro and in vivo. By synthesizing and screening a small library of 18 PBAE polymers with different backbone and end-group chemistry, we identified lead cationic PBAE structures that can complex with minicircle DNA to form nanoparticles, and delivery efficiency can be further modulated by tuning PBAE chemistry. Using human embryonic kidney 293 cells and mouse embryonic fibroblasts as model cell types, we identified a few PBAE polymers that allow efficient MC delivery at levels that are comparable or even surpassing Lipofectamine 2000. The biodegradable nature of PBAE-based nanoparticles facilitates in vivo applications and clinical translation. When injected via intraperitoneal route in vivo, MC alone resulted in high transgene expression, and a lead PBAE/MC nanoparticle formulation achieved a further 2-fold increase in protein expression compared to MC alone. Together, our results highlight the promise of PBAE-based nanoparticles as promising nonviral gene carriers for MC delivery, which may provide a valuable tool for broad applications of MC DNA-based gene therapy.

Indexed as

AnimalsBiocompatible MaterialsCationsCell SeparationCell SurvivalDNAFemaleFibroblastsFlow CytometryGenetic TherapyGenetic VectorsGreen Fluorescent ProteinsHEK293 CellsHumansMiceMicroscopy, Electron, TransmissionBiocompatible MaterialsCationsDNAGreen Fluorescent Proteinspoly(beta-amino ester)Polymers

Identifiers

PMID23837668
PMCPMC3789527
OpenAlexW2024409869

What OpenQuestion holds

Textmetadata
LicenceTDM
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.