Evidence map›Paper›PMID 27981708›Full record

ArticleAngewandte Chemie (International ed. in English)2017

Non-Viral CRISPR/Cas Gene Editing In Vitro and In Vivo Enabled by Synthetic Nanoparticle Co-Delivery of Cas9 mRNA and sgRNA.

Jason B Miller, Shuyuan Zhang, Petra Kos, Hu Xiong, Kejin Zhou, Sofya S Perelman, Hao Zhu, Daniel J Siegwart

Abstract read
In one paragraph

Article in Angewandte Chemie (International ed. in English), 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 244 papers.

0numbers the graph read from it
0cells of the map it votes in
244citing papers in PubMed
–field-weighted citation impact
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

244 citing papers in PubMed.

  1. Cas12a chRDNA-mediatedMolecular therapy. Nucleic acids · 2026
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  9. Messenger RNA and guide RNA distributions in lipid nanoparticles impact gene-editing efficiency in vivo.Molecular therapy : the journal of the American Society of Gene Therapy · 2026
    Article
  10. Article
  11. Article
  12. Review
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  15. Article
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  20. Review

184 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.

Jason B MillerUniversity of Texas Southwestern Medical Center, Simmons Comprehensive Cancer Center, Department of Biochemistry, Dallas, TX, 75390, USA.ORCID 0000-0002-6587-250X
Shuyuan ZhangUniversity of Texas Southwestern Medical Center, Children's Research Institute, Departments of Pediatrics and Internal Medicine, Dallas, TX, 75390, USA.
Petra KosUniversity of Texas Southwestern Medical Center, Simmons Comprehensive Cancer Center, Department of Biochemistry, Dallas, TX, 75390, USA.
Hu XiongUniversity of Texas Southwestern Medical Center, Simmons Comprehensive Cancer Center, Department of Biochemistry, Dallas, TX, 75390, USA.
Kejin ZhouUniversity of Texas Southwestern Medical Center, Simmons Comprehensive Cancer Center, Department of Biochemistry, Dallas, TX, 75390, USA.
Sofya S PerelmanUniversity of Texas Southwestern Medical Center, Department of Microbiology, Dallas, TX, 75390, USA.
Hao ZhuUniversity of Texas Southwestern Medical Center, Children's Research Institute, Departments of Pediatrics and Internal Medicine, Dallas, TX, 75390, USA.
Daniel J SiegwartUniversity of Texas Southwestern Medical Center, Simmons Comprehensive Cancer Center, Department of Biochemistry, Dallas, TX, 75390, USA.ORCID 0000-0003-3823-1931

Funding

Reactivation of embryonic growth programs in liver cancerR01CA190525 · NCI · UT SOUTHWESTERN MEDICAL CENTER · PI ZHU, HAO · 2015 to 2019
$1.8M
NCI NIH HHS R01 CA190525
6 · The paper itself

Abstract

CRISPR/Cas is a revolutionary gene editing technology with wide-ranging utility. The safe, non-viral delivery of CRISPR/Cas components would greatly improve future therapeutic utility. We report the synthesis and development of zwitterionic amino lipids (ZALs) that are uniquely able to (co)deliver long RNAs including Cas9 mRNA and sgRNAs. ZAL nanoparticle (ZNP) delivery of low sgRNA doses (15 nm) reduces protein expression by >90 % in cells. In contrast to transient therapies (such as RNAi), we show that ZNP delivery of sgRNA enables permanent DNA editing with an indefinitely sustained 95 % decrease in protein expression. ZNP delivery of mRNA results in high protein expression at low doses in vitro (<600 pM) and in vivo (1 mg kg

Indexed as

Gene EditingGene Transfer TechniquesCRISPR-Cas SystemsLipidsMolecular StructureNanoparticlesRNA, Guide, CRISPR-Cas SystemsRNA, MessengerLipidsRNA, Guide, CRISPR-Cas SystemsRNA, MessengerCRISPR/Casgene editingmRNA deliverynanoparticlessgRNA delivery

Identifiers

PMID27981708
PMCPMC5521011

What OpenQuestion holds

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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.