Evidence map›Paper›PMID 31840076›Full record

ArticleScience advances2019

Carboxylated branched poly(β-amino ester) nanoparticles enable robust cytosolic protein delivery and CRISPR-Cas9 gene editing.

Yuan Rui, David R Wilson, John Choi, Mahita Varanasi, Katie Sanders, Johan Karlsson, Michael Lim, Jordan J Green

Open access · goldAbstract read
In one paragraph

Article in Science advances, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 90 papers.

0numbers the graph read from it
0cells of the map it votes in
90citing papers in PubMed
7.7field-weighted citation impact, top 2% 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

90 citing papers in PubMed, 189 citations in OpenAlex.

  1. Review
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  7. Tissue-specific gene delivery approaches.Bioengineering & translational medicine · 2026
    Review
  8. Article
  9. Review
  10. Nanocarrier-mediated CRISPR-Cas delivery: a novel approach against antibiotic-resistant superbugs.Saudi pharmaceutical journal : SPJ : the official publication of the Saudi Pharmaceutical Society · 2026
    Review
  11. Review
  12. Article
  13. Article
  14. Review
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  20. Article

30 more citing papers are in PubMed but not listed here.

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 2 institutions in 1 country.

Yuan RuiDepartment of Biomedical Engineering, Institute for NanoBioTechnology, and Translational Tissue Engineering Center, Johns Hopkins University School of Medicine, Baltimore, MD, USA.ORCID 0000-0001-5165-8898
David R WilsonDepartment of Biomedical Engineering, Institute for NanoBioTechnology, and Translational Tissue Engineering Center, Johns Hopkins University School of Medicine, Baltimore, MD, USA.ORCID 0000-0002-8708-4248
John ChoiDepartment of Neurosurgery, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Mahita VaranasiDepartment of Biomedical Engineering, Institute for NanoBioTechnology, and Translational Tissue Engineering Center, Johns Hopkins University School of Medicine, Baltimore, MD, USA.ORCID 0000-0002-7931-1682
Katie SandersDepartment of Biomedical Engineering, Institute for NanoBioTechnology, and Translational Tissue Engineering Center, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Johan KarlssonDepartment of Biomedical Engineering, Institute for NanoBioTechnology, and Translational Tissue Engineering Center, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Michael LimDepartment of Neurosurgery, Johns Hopkins University School of Medicine, Baltimore, MD, USA.ORCID 0000-0003-0523-3076
Jordan J GreenDepartment of Biomedical Engineering, Institute for NanoBioTechnology, and Translational Tissue Engineering Center, Johns Hopkins University School of Medicine, Baltimore, MD, USA.ORCID 0000-0003-4176-3808
Johns Hopkins University · USBloomberg (United States) · US

Funding

Wilmer Core Grant for Vision ResearchP30EY001765 · NEI · JOHNS HOPKINS UNIVERSITY · PI Ji Yi · 1985 to 2026
$22.1M
BIOMEDICAL ENGINEERING TRAINING PROGRAMT32GM007057 · NIGMS · JOHNS HOPKINS UNIVERSITY · PI KANOLD, PATRICK O, KARCHIN, RACHEL · 1985 to 2023
$10.4M
Gene Delivery Nanoparticles to Treat GlioblastomaR01CA228133 · NCI · JOHNS HOPKINS UNIVERSITY · PI GREEN, JORDAN · 2019 to 2023
$1.9M
Cancer-specific nanoparticle-mediated gene therapy to treat hepatocellular carcinomaR01EB022148 · NIBIB · JOHNS HOPKINS UNIVERSITY · PI GREEN, JORDAN · 2016 to 2019
$1.5M
Zeiss LSM780 Confocal Microscope for a Core FacilityS10OD016374 · OD · JOHNS HOPKINS UNIVERSITY · PI KUO, SCOT CHARLES · 2013 to 2013
$584k
NCI NIH HHS R01 CA228133NEI NIH HHS P30 EY001765NIBIB NIH HHS R01 EB022148NIGMS NIH HHS T32 GM007057NIH HHS S10 OD016374
6 · The paper itself

Abstract

Efficient cytosolic protein delivery is necessary to fully realize the potential of protein therapeutics. Current methods of protein delivery often suffer from low serum tolerance and limited in vivo efficacy. Here, we report the synthesis and validation of a previously unreported class of carboxylated branched poly(β-amino ester)s that can self-assemble into nanoparticles for efficient intracellular delivery of a variety of different proteins. In vitro, nanoparticles enabled rapid cellular uptake, efficient endosomal escape, and functional cytosolic protein release into cells in media containing 10% serum. Moreover, nanoparticles encapsulating CRISPR-Cas9 ribonucleoproteins (RNPs) induced robust levels of gene knock-in (4%) and gene knockout (>75%) in several cell types. A single intracranial administration of nanoparticles delivering a low RNP dose (3.5 pmol) induced robust gene editing in mice bearing engineered orthotopic murine glioma tumors. This self-assembled polymeric nanocarrier system enables a versatile protein delivery and gene editing platform for biological research and therapeutic applications.

Indexed as

Gene Transfer TechniquesAnimalsCRISPR-Cas SystemsCytosolGene EditingGliomaHumansMiceNanoparticlesPolymersRibonucleoproteinspoly(beta-amino ester)PolymersRibonucleoproteins

Identifiers

PMID31840076
PMCPMC6897553
OpenAlexW2992326035

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.