Evidence map›Paper›PMID 37833442›Full record

ArticleNature communications2023

Lipid nanoparticles with PEG-variant surface modifications mediate genome editing in the mouse retina.

Milan Gautam, Antony Jozic, Grace Li-Na Su, Marco Herrera-Barrera, Allison Curtis, Sebastian Arrizabalaga, Wayne Tschetter, Renee C Ryals, Gaurav Sahay

Open access · goldAbstract read
In one paragraph

Article in Nature communications, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 62 papers.

0numbers the graph read from it
0cells of the map it votes in
62citing papers in PubMed
16.5field-weighted citation impact, top 1% 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

62 citing papers in PubMed, 107 citations in OpenAlex.

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  16. International journal of biological sciences · 2026
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2 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

9 authors at 2 institutions in 1 country.

Milan GautamDepartment of Pharmaceutical Sciences, College of Pharmacy, Robertson Life Sciences Building, Oregon State University, Portland, OR, 97201, USA.ORCID http://orcid.org/0000-0002-3817-620X
Antony JozicDepartment of Pharmaceutical Sciences, College of Pharmacy, Robertson Life Sciences Building, Oregon State University, Portland, OR, 97201, USA.ORCID http://orcid.org/0000-0001-6249-2328
Grace Li-Na SuDepartment of Ophthalmology, Casey Eye Institute, Oregon Health & Science University, Portland, OR, 97239, USA.ORCID http://orcid.org/0000-0001-9328-5620
Marco Herrera-BarreraDepartment of Pharmaceutical Sciences, College of Pharmacy, Robertson Life Sciences Building, Oregon State University, Portland, OR, 97201, USA.
Allison CurtisDepartment of Ophthalmology, Casey Eye Institute, Oregon Health & Science University, Portland, OR, 97239, USA.
Sebastian ArrizabalagaDepartment of Ophthalmology, Casey Eye Institute, Oregon Health & Science University, Portland, OR, 97239, USA.
Wayne TschetterDepartment of Ophthalmology, Casey Eye Institute, Oregon Health & Science University, Portland, OR, 97239, USA.
Renee C RyalsDepartment of Ophthalmology, Casey Eye Institute, Oregon Health & Science University, Portland, OR, 97239, USA. ryals@ohsu.edu.ORCID http://orcid.org/0000-0002-7182-7238
Gaurav SahayDepartment of Pharmaceutical Sciences, College of Pharmacy, Robertson Life Sciences Building, Oregon State University, Portland, OR, 97201, USA. sahay@ohsu.edu.ORCID http://orcid.org/0000-0003-1071-0500
Oregon Health & Science University · USOregon State University · US

Funding

Proteomics CoreP30EY010572 · NEI · OREGON HEALTH & SCIENCE UNIVERSITY · PI John Peter Campbell · 1995 to 2026
$19.4M
Program in Biomedical SciencesT32GM142619 · NIGMS · OREGON HEALTH & SCIENCE UNIVERSITY · PI Jeffrey Wallace Tyner · 2021 to 2026
$3.2M
Developing gene editing platforms for retinal degeneration.R01EY033423 · NEI · OREGON STATE UNIVERSITY · PI Gaurav Sahay · 2022 to 2026
$3.1M
NEI NIH HHS P30 EY010572NEI NIH HHS R01 EY033423NIGMS NIH HHS T32 GM142619
6 · The paper itself

Abstract

Ocular delivery of lipid nanoparticle (LNPs) packaged mRNA can enable efficient gene delivery and editing. We generated LNP variants through the inclusion of positively charged-amine-modified polyethylene glycol (PEG)-lipids (LNPa), negatively charged-carboxyl-(LNPz) and carboxy-ester (LNPx) modified PEG-lipids, and neutral unmodified PEG-lipids (LNP). Subretinal injections of LNPa containing Cre mRNA in the mouse show tdTomato signal in the retinal pigmented epithelium (RPE) like conventional LNPs. Unexpectedly, LNPx and LNPz show 27% and 16% photoreceptor transfection, respectively, with striking localization extending from the photoreceptor synaptic pedicle to the outer segments, displaying pan-retinal distribution in the photoreceptors and RPE. LNPx containing Cas9 mRNA and sgAi9 leads to the formation of an oval elongated structure with a neutral charge resulting in 16.4% editing restricted to RPE. Surface modifications of LNPs with PEG variants can alter cellular tropism of mRNA. LNPs enable genome editing in the retina and in the future can be used to correct genetic mutations that lead to blindness.

Indexed as

NanoparticlesPolyethylene GlycolsAnimalsGene EditingLipidsLiposomesMiceRetinal Pigment EpitheliumRNA, MessengerRNA, Small InterferingLipid NanoparticlesLipidsLiposomesPolyethylene GlycolsRNA, MessengerRNA, Small Interfering

Identifiers

PMID37833442
PMCPMC10575971
OpenAlexW4387609025

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.