Evidence map›Paper›PMID 41654520›Full record

ArticleNature communications2026

A combinatorial synthetic strategy for developing genome-editing protein-delivery agents targeting mouse retina.

Jianye Zhang, Rafał Hołubowicz, Roman Smidak, Yulun Hu, Samuel W Du, Jiin H Felgner, Grazyna Palczewska, Carolline Rodrigues Menezes, Eleonora Risaliti, Zhiqian Dong and 10 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Nature communications, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. Article
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

20 authors.

Jianye Zhang *Gavin Herbert Eye Institute - Brunson Center for Translational Vision Research, Department of Ophthalmology, University of California, Irvine, Irvine, CA, USA.
Rafał Hołubowicz *Gavin Herbert Eye Institute - Brunson Center for Translational Vision Research, Department of Ophthalmology, University of California, Irvine, Irvine, CA, USA.
Roman Smidak *Gavin Herbert Eye Institute - Brunson Center for Translational Vision Research, Department of Ophthalmology, University of California, Irvine, Irvine, CA, USA.
Yulun HuDepartment of Chemistry, College of Arts and Sciences, Case Western Reserve University, Cleveland, OH, USA.ORCID 0000-0002-2181-1182
Samuel W DuGavin Herbert Eye Institute - Brunson Center for Translational Vision Research, Department of Ophthalmology, University of California, Irvine, Irvine, CA, USA.ORCID 0000-0002-9655-3659
Jiin H FelgnerAdeline Yen Mah Vaccine Center, Department of Physiology and Biophysics, University of California, Irvine, California, USA.
Grazyna PalczewskaGavin Herbert Eye Institute - Brunson Center for Translational Vision Research, Department of Ophthalmology, University of California, Irvine, Irvine, CA, USA.
Carolline Rodrigues MenezesGavin Herbert Eye Institute - Brunson Center for Translational Vision Research, Department of Ophthalmology, University of California, Irvine, Irvine, CA, USA.
Eleonora RisalitiGavin Herbert Eye Institute - Brunson Center for Translational Vision Research, Department of Ophthalmology, University of California, Irvine, Irvine, CA, USA.ORCID 0009-0006-9689-7474
Zhiqian DongGavin Herbert Eye Institute - Brunson Center for Translational Vision Research, Department of Ophthalmology, University of California, Irvine, Irvine, CA, USA.ORCID 0000-0002-8748-4532
Xiuli MaGavin Herbert Eye Institute - Brunson Center for Translational Vision Research, Department of Ophthalmology, University of California, Irvine, Irvine, CA, USA.
Mojtaba H ShayeganDepartment of Chemistry, College of Arts and Sciences, Case Western Reserve University, Cleveland, OH, USA.
Paul Z ChenMerkin Institute for Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA, USA.
Li XingUC Irvine Materials Research Institute, University of California, Irvine, Irvine, CA, USA.ORCID 0000-0001-7612-602X
Maria HołubowiczGavin Herbert Eye Institute - Brunson Center for Translational Vision Research, Department of Ophthalmology, University of California, Irvine, Irvine, CA, USA.
Bowen LiDepartment of Chemistry, College of Arts and Sciences, Case Western Reserve University, Cleveland, OH, USA.
David R LiuMerkin Institute for Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA, USA.
Philip L FelgnerAdeline Yen Mah Vaccine Center, Department of Physiology and Biophysics, University of California, Irvine, California, USA.
Gregory P TochtropDepartment of Chemistry, College of Arts and Sciences, Case Western Reserve University, Cleveland, OH, USA. gpt6@case.edu.ORCID 0000-0003-2447-254X
Krzysztof PalczewskiGavin Herbert Eye Institute - Brunson Center for Translational Vision Research, Department of Ophthalmology, University of California, Irvine, Irvine, CA, USA. kpalczew@uci.edu.ORCID 0000-0002-0788-545X

Funding

Univ.of Calif., Irvine Cancer Center Support GrantP30CA062203 · NCI · UNIVERSITY OF CALIFORNIA-IRVINE · PI Melanie Funes · 1994 to 2026
$57.9M
Center for Genomic Editing and Recording: Development and Application of Next-Generation Genome and Epigenome Editing Methods to Advance the Study and Treatment of Human DiseaseRM1HG009490 · NHGRI · WHITEHEAD INSTITUTE FOR BIOMEDICAL RES · PI Brittany S. Adamson, Martin Joseph Ankrah Aryee · 2017 to 2026
$22.7M
STRUCTURAL STUDIES OF ARRESTINSR01EY009339 · NEI · UNIVERSITY OF WASHINGTON · PI KISER, PHILIP DAVID, PALCZEWSKI, KRZYSZTOF · 1992 to 2025
$16.0M
Adjuvant Comparison and Characterization in Influenza , Chlamydia muridarum, and Coxiella burnetii Vaccines75N93022C00054 · NIAID · UNIVERSITY OF CALIFORNIA-IRVINE · PI FELGNER, PHILIP · 2022 to 2025
$10.2M
MEDICAL SCIENTIST TRAINING PROGRAMT32GM008620 · NIGMS · UNIVERSITY OF CALIFORNIA-IRVINE · PI GOLDIN, ALAN L · 1999 to 2023
$8.1M
Integrating Chemistry and Evolution to Illuminate Biology and Enable Novel TherapeuticsR35GM118062 · NIGMS · HARVARD UNIVERSITY · PI LIU, DAVID R · 2016 to 2025
$6.4M
Precision genome editing in vivo to treat retinal diseasesR01EY034501 · NEI · UNIVERSITY OF CALIFORNIA-IRVINE · PI Audrone Lapinaite, Krzysztof Palczewski · 2023 to 2026
$3.9M
NEI UCI Center Core Grant for Vision ResearchP30EY034070 · NEI · UNIVERSITY OF CALIFORNIA-IRVINE · PI Vladimir Jivkov Kefalov · 2022 to 2026
$3.7M
The complex role of phosphodiesterase 6 in rod photoreceptor health and functionR01EY030873 · NEI · UNIVERSITY OF CALIFORNIA-IRVINE · PI Krzysztof Palczewski · 2020 to 2026
$3.5M
Delivery Technologies for In Vivo Genome EditingUG3AI150551 · NIAID · BETH ISRAEL DEACONESS MEDICAL CENTER · PI CHAIKOF, ELLIOT · 2019 to 2021
$2.3M
Expanding the Scope of Base EditingU01AI142756 · NIAID · BROAD INSTITUTE, INC. · PI LIU, DAVID R · 2018 to 2022
$2.1M
microRNA-204 and microRNA-211 regulation of RPE phagocytosisF30EY033642 · NEI · UNIVERSITY OF CALIFORNIA-IRVINE · PI Samuel Wang Du · 2022 to 2026
$241k
Howard Hughes Medical InstituteKnights Templar Eye FoundationNational Science Foundation (NSF) CHE-1904530NCI NIH HHS P30 CA062203NEI NIH HHS F30 EY033642NEI NIH HHS P30 EY034070NEI NIH HHS R01 EY009339NEI NIH HHS R01 EY030873NEI NIH HHS R01 EY034501NHGRI NIH HHS RM1 HG009490NIAID NIH HHS U01 AI142756NIAID NIH HHS UG3 AI150551NIGMS NIH HHS R35 GM118062NIGMS NIH HHS T32 GM008620NIH HHS 75N93022C00054University of California, Irvine School of MedicineU.S. Department of Health & Human Services | NIH | National Eye Institute (NEI) R01EY009339; R01EY030873, P30EY034070U.S. Department of Health & Human Services | NIH | National Eye Institute (NEI) T32GM008620; F30EY033642U.S. Department of Health & Human Services | NIH | NCI | Division of Cancer Epidemiology and Genetics, National Cancer Institute (National Cancer Institute Division of Cancer Epidemiology and Genetics) P30CA062203
6 · The paper itself

Abstract

CRISPR/Cas9-based gene-editing technologies offer promise for treating inherited retinal diseases (IRDs), however safe and efficient ocular delivery of precision editors remains challenging. To address this challenge, we report a class of Coomassie brilliant blue (CBB)-derived lipidoids that bind and deliver proteins. Subretinal injection of Cre complexed with these lipidoids into mT/mG mice leads to robust recombination in the retinal pigment epithelium and photoreceptors. We employ the CBB-lipidoid platform to deliver adenine base editor (ABE) ribonucleoproteins (RNP). Incorporating CBB lipidoids into liposomes improves delivery efficiency. CBB11 stands out for facilitating precise in vivo ABE-mediated gene editing. Delivery of liposome-CBB11-RNP complexes results in a 120-fold increase in base editing compared to RNP alone and restores the scotopic ERG b-wave response in the rd12 mouse model. These results demonstrate the potential of CBB-augmented, liposome-RNP systems for therapeutic gene editing in the eye, paving the way for single-dose precision medicines to treat IRDs.

Indexed as

Gene EditingRetinaAnimalsCRISPR-Cas SystemsDisease Models, AnimalElectroretinographyFemaleLiposomesMaleMiceRetinal Pigment EpitheliumRibonucleoproteinsRosaniline Dyescoomassie Brilliant BlueLiposomesRibonucleoproteinsRosaniline Dyes

Identifiers

PMID41654520
PMCPMC12992809

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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.