Evidence map›Paper›PMID 39921871›Full record

ArticleAdvanced science (Weinheim, Baden-Wurttemberg, Germany)2025

A Serum Resistant Polymer with Exceptional Endosomal Escape and mRNA Delivery Efficacy for CRISPR Gene Therapy.

Jia Lv, Qianqian Fan, Yirou Zhang, Xujiao Zhou, Panting Yu, Xin Yu, Changchang Xin, Jiaxu Hong, Yiyun Cheng

Abstract read
In one paragraph

Article in Advanced science (Weinheim, Baden-Wurttemberg, Germany), 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing 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

6 citing papers in PubMed.

  1. Polymeric Gene Delivery at Sub-Stoichiometric N/P Ratios.Polymer science & technology (Washington, D.C.) · 2026
    Article
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  4. Review
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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.

Jia LvShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, School of Life Sciences, East China Normal University, Shanghai, 200241, China.ORCID https://orcid.org/0009-0009-6390-6452
Qianqian FanShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, School of Life Sciences, East China Normal University, Shanghai, 200241, China.
Yirou ZhangDepartment of Ophthalmology and Vision Science, Eye, Ear, Nose, and Throat Hospital, Fudan University, Shanghai, 200030, China.
Xujiao ZhouDepartment of Ophthalmology and Vision Science, Eye, Ear, Nose, and Throat Hospital, Fudan University, Shanghai, 200030, China.
Panting YuShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, School of Life Sciences, East China Normal University, Shanghai, 200241, China.
Xin YuShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, School of Life Sciences, East China Normal University, Shanghai, 200241, China.
Changchang XinDepartment of Ophthalmology and Vision Science, Eye, Ear, Nose, and Throat Hospital, Fudan University, Shanghai, 200030, China.
Jiaxu HongDepartment of Ophthalmology and Vision Science, Eye, Ear, Nose, and Throat Hospital, Fudan University, Shanghai, 200030, China.
Yiyun ChengShanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, School of Life Sciences, East China Normal University, Shanghai, 200241, China.ORCID https://orcid.org/0000-0001-6019-5645

Funding

National Key R&D Program of China 2019YFA0904500National Key R&D Program of China 2023YFA0915000National Key Research and Development Program of China 2019YFA0904500National Key Research and Development Program of China 2023YFA0915000National Natural Science Foundation of China 52373134National Natural Science Foundation of China 82171102National Natural Science Foundation of China 82271044Shanghai Medical Innovation Research Program 22Y21900900
6 · The paper itself

Abstract

Nanoparticle-based mRNA delivery offers a versatile platform for innovative therapies. However, most of the current delivery systems are limited by poor serum tolerance, suboptimal endosomal escape and mRNA delivery efficacy. Herein, a highly efficient mRNA-delivering material is identified from a library of fluoropolymers. The lead material FD17 shows exceptional serum stability and endosomal escape, enabling efficient mRNA delivery into various cell types, surpassing commercial mRNA delivery reagents such as Lipofectamine 3000. The formed mRNA nanoparticles adsorb abundant serum albumin on the surface, which facilitates cellular uptake via scavenger receptor-mediated endocytosis. FD17 enables the delivery of mRNAs encoding CRE, Cas9, and base editor hyCBE for efficient genome editing. The material mediates CRISPR/Cas9 gene therapy via intraocular injection effectively down-regulates vascular endothelial growth factor A in retinal pigment epithelial cells of mice, yielding promising therapeutic responses against laser-induced choroidal neovascularization. The discovered material in this study shows great promise for the development of mRNA therapeutics to combat a wide range of diseases.

Indexed as

CRISPR-Cas SystemsEndosomesGenetic TherapyGene Transfer TechniquesPolymersRNA, MessengerAnimalsChoroidal NeovascularizationGene EditingHumansMiceNanoparticlesPolymersRNA, MessengerCRISPR gene therapyfluoropolymermRNA deliverynanoparticlepolymer

Identifiers

PMID39921871
PMCPMC11967772

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.