Evidence map›Paper›PMID 34174352›Full record

ArticleJournal of controlled release : official journal of the Controlled Release Society2021

In vivo targeted delivery of nucleic acids and CRISPR genome editors enabled by GSH-responsive silica nanoparticles.

Yuyuan Wang, Pawan K Shahi, Xiuxiu Wang, Ruosen Xie, Yi Zhao, Min Wu, Seth Roge, Bikash R Pattnaik, Shaoqin Gong

Open access · greenAbstract read
In one paragraph

Article in Journal of controlled release : official journal of the Controlled Release Society, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 48 papers.

0numbers the graph read from it
0cells of the map it votes in
48citing papers in PubMed
4.9field-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

48 citing papers in PubMed, 95 citations in OpenAlex.

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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

9 authors at 3 institutions in 1 country.

Yuyuan WangDepartment of Biomedical Engineering, University of Wisconsin-Madison, Madison, WI 53715, USA; Wisconsin Institute for Discovery, University of Wisconsin-Madison, Madison, WI 53715, USA.
Pawan K ShahiDepartment of Pediatrics, University of Wisconsin-Madison, Madison, WI 53706, USA; McPherson Eye Research Institute, University of Wisconsin-Madison, Madison, WI 53705, USA.
Xiuxiu WangDepartment of Biomedical Engineering, University of Wisconsin-Madison, Madison, WI 53715, USA; Wisconsin Institute for Discovery, University of Wisconsin-Madison, Madison, WI 53715, USA.
Ruosen XieWisconsin Institute for Discovery, University of Wisconsin-Madison, Madison, WI 53715, USA; Department of Materials Science and Engineering, University of Wisconsin-Madison, Madison, WI 53715, USA.
Yi ZhaoDepartment of Biomedical Engineering, University of Wisconsin-Madison, Madison, WI 53715, USA; Wisconsin Institute for Discovery, University of Wisconsin-Madison, Madison, WI 53715, USA.
Min WuDepartment of Biomedical Engineering, University of Wisconsin-Madison, Madison, WI 53715, USA.
Seth RogeDepartment of Biomedical Engineering, University of Wisconsin-Madison, Madison, WI 53715, USA.
Bikash R PattnaikDepartment of Pediatrics, University of Wisconsin-Madison, Madison, WI 53706, USA; McPherson Eye Research Institute, University of Wisconsin-Madison, Madison, WI 53705, USA; Department of Ophthalmology and Visual Sciences, University of Wisconsin-Madison, Madison, WI 53706, USA.
Shaoqin GongDepartment of Biomedical Engineering, University of Wisconsin-Madison, Madison, WI 53715, USA; Wisconsin Institute for Discovery, University of Wisconsin-Madison, Madison, WI 53715, USA; McPherson Eye Research Institute, University of Wisconsin-Madison, Madison, WI 53705, USA; Department of Materials Science and Engineering, University of Wisconsin-Madison, Madison, WI 53715, USA; Department of Chemistry, University of Wisconsin-Madison, Madison, WI 53715, USA. Electronic address: shaoqingong@wisc.edu.
University of Wisconsin–Madison · USSmith-Kettlewell Eye Research Institute · USWisconsin Institutes for Discovery · US

Funding

Restoring Vision with High-Fidelity Nonsense Codon CorrectionR24EY032434 · NEI · UNIVERSITY OF WISCONSIN-MADISON · PI AHERN, CHRISTOPHER A, GAMM, DAVID M · 2021 to 2025
$7.8M
Targeting PERK: An Endothelium-Protective Stent-Free Strategy for Mitigation of Intimal Hyperplasia After Vascular SurgeryR01HL143469 · NHLBI · UNIVERSITY OF VIRGINIA · PI GONG, SHAOQIN -, GUO, LIANWANG · 2018 to 2021
$2.4M
Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the BrainUG3NS111688 · NINDS · UNIVERSITY OF WISCONSIN-MADISON · PI EMBORG, MARINA, GONG, SHAOQIN - · 2018 to 2020
$2.3M
Development of unimolecular nanoparticle-mediated periadventitial drug delivery system for sustained and targeted inhibition of intimal hyperplasia following open vascular reconstructionR01HL129785 · NHLBI · UNIVERSITY OF WISCONSIN-MADISON · PI GONG, SHAOQIN -, GUO, LIANWANG · 2016 to 2019
$2.0M
Molecular Therapies for Lebers Congenital Amaurosis Caused by KCNJ13 MutationsR01EY024995 · NEI · UNIVERSITY OF WISCONSIN-MADISON · PI PATTNAIK, BIKASH RANJAN · 2015 to 2018
$1.5M
NEI NIH HHS R01 EY024995NEI NIH HHS R24 EY032434NHLBI NIH HHS R01 HL129785NHLBI NIH HHS R01 HL143469NINDS NIH HHS UG3 NS111688
6 · The paper itself

Abstract

The rapid development of gene therapy and genome editing techniques brings up an urgent need to develop safe and efficient nanoplatforms for nucleic acids and CRISPR genome editors. Herein we report a stimulus-responsive silica nanoparticle (SNP) capable of encapsulating biomacromolecules in their active forms with a high loading content and loading efficiency as well as a well-controlled nanoparticle size (~50 nm). A disulfide crosslinker was integrated into the silica network, endowing SNP with glutathione (GSH)-responsive cargo release capability when internalized by target cells. An imidazole-containing component was incorporated into the SNP to enhance the endosomal escape capability. The SNP can deliver various cargos, including nucleic acids (e.g., DNA and mRNA) and CRISPR genome editors (e.g., Cas9/sgRNA ribonucleoprotein (RNP), and RNP with donor DNA) with excellent efficiency and biocompatibility. The SNP surface can be PEGylated and functionalized with different targeting ligands. In vivo studies showed that subretinally injected SNP conjugated with all-trans-retinoic acid (ATRA) and intravenously injected SNP conjugated with GalNAc can effectively deliver mRNA and RNP to murine retinal pigment epithelium (RPE) cells and liver cells, respectively, leading to efficient genome editing. Overall, the SNP is a promising nanoplatform for various applications including gene therapy and genome editing.

Indexed as

NanoparticlesSilicon DioxideAnimalsCRISPR-Cas SystemsGene EditingGlutathioneMiceRNA, MessengerGlutathioneRNA, MessengerSilicon DioxideCRISPR-Cas9 genome editingGene deliverySilica nanoparticle

Identifiers

PMID34174352
PMCPMC8383466
OpenAlexW3173831391

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.