Evidence map›Paper›PMID 42806111›Full record

ArticleNature biotechnology2026

Lipid nanoparticles optimized for large RNA cargo and tissue targeting enhance in vivo genome editing.

Songtao Dong, Fanglin Gong, Tyler Thomson, Yunshu Cai, Lauren Healy, Rick X Z Lu, Ziyu Zhou, Yue Xu, Jingan Chen, Margarita Savguira and 6 more

Abstract read
PubMed Publisher
In one paragraph

Article in Nature biotechnology, 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

16 authors.

Songtao Dong *Leslie Dan Faculty of Pharmacy, University of Toronto, Toronto, Ontario, Canada.ORCID http://orcid.org/0009-0000-6841-6924
Fanglin Gong *Institute of Biomedical Engineering, University of Toronto, Toronto, Ontario, Canada.
Tyler Thomson *Institute of Biomedical Engineering, University of Toronto, Toronto, Ontario, Canada.ORCID http://orcid.org/0000-0002-1434-1813
Yunshu CaiLeslie Dan Faculty of Pharmacy, University of Toronto, Toronto, Ontario, Canada.
Lauren HealyDepartment of Chemistry, University of Toronto, Toronto, Ontario, Canada.ORCID http://orcid.org/0009-0003-5772-3269
Rick X Z LuLeslie Dan Faculty of Pharmacy, University of Toronto, Toronto, Ontario, Canada.
Ziyu ZhouLeslie Dan Faculty of Pharmacy, University of Toronto, Toronto, Ontario, Canada.ORCID http://orcid.org/0009-0004-8704-1952
Yue XuLeslie Dan Faculty of Pharmacy, University of Toronto, Toronto, Ontario, Canada.
Jingan ChenInstitute of Biomedical Engineering, University of Toronto, Toronto, Ontario, Canada.ORCID http://orcid.org/0009-0002-5105-102X
Margarita SavguiraInstitute of Biomedical Engineering, University of Toronto, Toronto, Ontario, Canada.
Xianglei FuLeslie Dan Faculty of Pharmacy, University of Toronto, Toronto, Ontario, Canada.
Sijin LuozhongLeslie Dan Faculty of Pharmacy, University of Toronto, Toronto, Ontario, Canada.
Muye ZhouLeslie Dan Faculty of Pharmacy, University of Toronto, Toronto, Ontario, Canada.ORCID http://orcid.org/0009-0008-8827-0609
Payton KirtleyVaccine and Gene therapy Institute, Oregon Health and Science University, Beaverton, OR, USA.
Brandon K WilderVaccine and Gene therapy Institute, Oregon Health and Science University, Beaverton, OR, USA.
Bowen LiLeslie Dan Faculty of Pharmacy, University of Toronto, Toronto, Ontario, Canada. bw.li@utoronto.ca.ORCID http://orcid.org/0000-0001-5006-9143

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Although lipid nanoparticles are clinically validated for RNA delivery, their potency often declines as transcript size increases. Here we report a large-cargo-informed lipid discovery strategy that incorporates mRNA size into ionizable lipid screening. A combinatorial library of 384 lipids was screened using a 5.7-kb ABE-NanoLuc reporter mRNA, identifying large-cargo-optimized lipids led by LC-1. Following intravenous, intrathecal and intratracheal administration, LC-1 achieved up to 79% Cas9-mediated knockout in liver and 48% and 27% in brain and lung, up to fourfold higher than LP-01 and ALC-0315, and produced higher ABE-mediated reporter correction across all three routes. LC-1 supported base editing of PCSK9, CFTR

Identifiers

PMID42806111

What OpenQuestion holds

Textmetadata
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.