Evidence map›Paper›PMID 41223249›Full record

ArticleScience translational medicine2025

Extracellular vesicle-mediated gene editing for the treatment of nonsyndromic progressive hearing loss in adult mice.

Xiaoshu Pan, Peixin Huang, Samantha S Ali, Bryan Renslo, Zachary Greenberg, Nina Erwin, Yanjun Li, Zuo Ding, Tarun E Hutchinson, Athanasia Warnecke and 3 more

Erratum issuedAbstract read
In one paragraph

Article in Science translational medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing 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

5 citing papers in PubMed.

  1. Review
  2. Toward an age of CRISPR delivery with non-viral biologics.Molecular therapy. Nucleic acids · 2026
    Article
  3. Review
  4. Review
  5. Advancing precision ear medicine: leveraging animal models for disease insights and therapeutic innovations.Mammalian genome : official journal of the International Mammalian Genome Society · 2025
    Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

13 authors.

Xiaoshu PanCollege of Pharmacy, Department of Pharmaceutics, University of Florida, Gainesville, FL 32611, USA.ORCID 0000-0003-1896-2198
Peixin HuangDepartment of Otolaryngology, Head and Neck Surgery, University of Kansas School of Medicine, Kansas City, KS 66160, USA.
Samantha S AliCollege of Pharmacy, Department of Pharmaceutics, University of Florida, Gainesville, FL 32611, USA.ORCID 0009-0004-4582-1678
Bryan RensloDepartment of Otolaryngology, Head and Neck Surgery, University of Kansas School of Medicine, Kansas City, KS 66160, USA.
Zachary GreenbergCollege of Pharmacy, Department of Pharmaceutics, University of Florida, Gainesville, FL 32611, USA.ORCID 0000-0001-9626-2694
Nina ErwinCollege of Pharmacy, Department of Pharmaceutics, University of Florida, Gainesville, FL 32611, USA.ORCID 0000-0002-2692-3714
Yanjun LiDepartment of Medicinal Chemistry, Center for Natural Products, Drug Discovery, and Development, University of Florida, Gainesville, FL 32610, USA.ORCID 0000-0002-6277-4189
Zuo DingCollege of Pharmacy, Department of Pharmaceutics, University of Florida, Gainesville, FL 32611, USA.ORCID 0000-0002-5901-4621
Tarun E HutchinsonCollege of Pharmacy, Department of Pharmaceutics, University of Florida, Gainesville, FL 32611, USA.ORCID 0000-0002-9175-7222
Athanasia WarneckeDepartment of Otolaryngology, Hannover Medical School, 30625 Hannover, Germany.
Natalia E FernandezCollege of Pharmacy, Department of Pharmaceutics, University of Florida, Gainesville, FL 32611, USA.
Hinrich StaeckerDepartment of Otolaryngology, Head and Neck Surgery, University of Kansas School of Medicine, Kansas City, KS 66160, USA.ORCID 0000-0002-0348-3015
Mei HeCollege of Pharmacy, Department of Pharmaceutics, University of Florida, Gainesville, FL 32611, USA.ORCID 0000-0001-5316-9326

Funding

Equipment Supplement to R35GM133794R35GM133794 · NIGMS · UNIVERSITY OF KANSAS LAWRENCE · PI Mei He · 2019 to 2026
$2.9M
Evaluation of High-throughput Extracellular Vesicle Loading Platform for Therapeutic Genome Editing INDsU01AI191208 · NIAID · UNIVERSITY OF FLORIDA · PI Mei He · 2025 to 2026
$1.4M
NIAID NIH HHS U01 AI191208NIGMS NIH HHS R35 GM133794
6 · The paper itself

Abstract

The clinical translation of gene therapy has been challenging in part because of the limitations of current delivery approaches. Herein, we report an efficient nonviral genome editor delivery approach using extracellular vesicles (EVs) carrying single-guide RNA (sgRNA): CRISPR-Cas9 ribonucleoprotein (RNP) complexes for in vivo gene therapy. By leveraging a high-throughput microfluidic droplet-based electroporation system (μDES), we achieved a 10-fold enhancement in loading efficiency and more than 1000-fold increase in processing throughput for loading RNP complexes into EVs compared with conventional high-voltage pulsed electroporation. μDES generated uniform microdroplets containing EVs and RNPs by applying direct current-controlled low voltage (up to 60 V) to transiently permeabilize membranes and enable efficient cargo encapsulation while maintaining EV integrity at both the protein and morphological levels. In the

Indexed as

Extracellular VesiclesGene EditingHearing LossAnimalsCRISPR-Cas SystemsDisease Models, AnimalElectroporationGenetic TherapyHair Cells, AuditoryHumansMiceRibonucleoproteinsRNA, Guide, CRISPR-Cas SystemsRibonucleoproteinsRNA, Guide, CRISPR-Cas Systems

Identifiers

PMID41223249
PMCPMC13051476

What OpenQuestion holds

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Registered trials

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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.