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RESEARCH / LITERATURE

Research Literature

Find research, methods, and reviews across our scientific fields.

One collection · Original publication dates · Authors, journals & DOIs

All publications 147

Duplicate DOIs are merged. This is a selected collection, not an exhaustive bibliography.

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Methods
Journal of the American Chemical Society

Photochemistry-Based Development of Ionizable Lipid Library for mRNA-Lipid Nanoparticle Delivery In Vivo

Simin Chun; Hyorim Nam; Donghyun Lee; Yeeun Lee; Cheol Hui Park; Chaehwan Lim; Donghyun Kim; Ji Sun Park; Kee-Pyo Kim; Hak Soo Choi; Heebeom Koo

Visible-light-driven multicomponent synthesis generated 275 branched amine-containing ionizable lipids from modular building blocks. The platform linked one-pot synthesis with purification-free cell screening and subsequent animal evaluation. A lead lipid improved expression relative to the tested MC3 formulation, and formulation adjustment enabled organ-selective delivery. The work accelerates discovery but does not establish a clinically optimized or validated manufacturing process.

mRNA / LNP
Read guide →Original paper ↗

DOI: 10.1021/jacs.6c13674

Reviews & perspectives
The Journal of Clinical Pharmacology

Model-Informed Decision Making from Drug Discovery to Reimbursement: A Roadmap for Clinical Pharmacology in Value-Based Healthcare

Rajesh Krishna; Ananth Kadambi; Patrick Smith; Piet H van der Graaf; Fran Brown

This commentary proposes a lifecycle approach to model-informed decision-making, from discovery and dose selection to regulatory evaluation and reimbursement. It discusses quantitative methods, patient-relevant outcomes, and real-world data, alongside challenges in integration, transparency, and stakeholder acceptance. It is a conceptual roadmap, not a prospective validation of one modeling strategy.

Clinical Pharmacology & Model-Informed Development
Read guide →Original paper ↗

DOI: 10.1002/jcph.70270

Original research
Cell Biomaterials

A branched ionizable lipid nanoparticle platform for versatile in vivo delivery of nucleic acids: Validation from mRNA to capped circular RNA

Seigo Kimura; Susumu Tsutsumi; Naoto Nakamura; Shigetomo Tsujihata; Lyu Fangjie; Yuko Nakashima; Shunsuke Kita; Katsumi Maenaka; Fumitaka Hashiya; Masahito Inagaki; Naoko Abe; Yasuaki Kimura; Hiroshi Abe

The branched ionizable lipid FL0445 supported delivery of linear mRNA and capped circular RNA through a cholesterol-dependent cellular-entry pathway. In obese mice, GLP-1-encoding capped circular RNA produced sustained expression and improved glucose tolerance relative to tested formulations. Inflammatory transcriptional signals were also assessed. The results provide a preclinical delivery proof of concept, not evidence of clinical metabolic efficacy.

mRNA / LNP
Read guide →Original paper ↗

DOI: 10.1016/j.celbio.2026.100555

Original research
ACS Nano

2-arm-PEG-Oligocations Transiently Shield the Liver Sinusoids to Mitigate Off-Target Hepatic Expression of mRNA Lipid Nanoparticles

Anjaneyulu Dirisala; Bhaskar Chatterjee; Nguyen Bui Thao Le; Kazuko Toh; Miki Matsui-Masai; Saed Abbasi; Xueying Liu; Theofilus A. Tockary; Nan Qiao; Junichi Ishikawa; Jumpei Norimatsu; Yuki Mochida; Shigeto Fukushima; Makoto Oba; Kazunori Kataoka; Satoshi Uchida

Researchers used two-armed PEG conjugated to oligocations to transiently coat liver sinusoidal endothelium before mRNA-LNP exposure. The approach modifies the host delivery barrier rather than the LNP itself, aiming to reduce hepatic entry and off-target expression. Mouse experiments examined reversible coating and clearance. Translation would require evaluating the combined regimen, vascular effects, and repeat-dose safety.

mRNA / LNP
Read guide →Original paper ↗

DOI: 10.1021/acsnano.6c08403

Original research
Proceedings of the National Academy of Sciences

Overcoming the blood–brain barrier using central nervous system–accessing lipid nanoparticles for enhanced mRNA therapeutics

Siyu Wang; Yichen Zhong; Chang Wang; Meng Tian; Jeffrey L. Bennett; Eliza Bliss-Moreau; Yonger Xue; Changyue Yu; Xucheng Hou; Ya Ying Zheng; Haoyuan Li; Zhengwei Liu; Dinglingge Cao; Diana D. Kang; Binbin Deng; Yizhou Dong

The CA2d ionizable-LNP platform delivered mRNA to brain cells after intravenous administration in mice and nonhuman primates. Reported target populations included neurons, microglia, and astrocytes. In a rat ischemic-stroke model, combined thrombolytic and neuroprotective mRNA cargos reduced injury and improved functional measures. The work expands preclinical CNS delivery evidence without demonstrating human brain delivery or treatment efficacy.

mRNA / LNP
Read guide →Original paper ↗

DOI: 10.1073/pnas.2538147123

Original research
Nano Letters

Enhancing Lipid Nanoparticle-Mediated Circular RNA and mRNA Expression in the Placenta through Inhibition of IFNAR-JAK-STAT Signaling

Hannah C. Safford; Hannah C. Geisler; Ajay S. Thatte; Kuo-Chieh Liao; Alexandre Poirier; Marshall Padilla; Hannah M. Yamagata; Anushka Agrawal; Amanda M. Murray; Alex G. Hamilton; Kelsey L. Swingle; Emily Fitzgerald; Ori Chalom; Yue Wan; Michael J. Mitchell

Placenta-tropic LNPs produced strong circular-RNA expression in cultured trophoblasts but weaker translation in pregnant mice than linear mRNA. Researchers identified innate immune signaling as a constraint and increased placental expression by inhibiting IFNAR-JAK-STAT pathways. VEGF-encoding cargos provided a therapeutic-payload demonstration. Immune suppression during pregnancy raises separate safety questions that expression improvements alone cannot resolve.

mRNA / LNP
Read guide →Original paper ↗

DOI: 10.1021/acs.nanolett.6c01762

Methods
Nature Communications

pH-Sensitive Amino Lipid-Driven Pore Formation Enables Endosomal Escape of Lipid Nanoparticles

Akhil Pratap Singh; Kana Shibata; Yusuke Miyazaki; Wataru Shinoda

Molecular simulations examined how ionizable lipid nanoparticles interact with endosomal membranes and release nucleic acids. The authors identified several fusion routes, with a stalk-pore pathway predominating, and linked release to protonation, lipid geometry, membrane tension, and cargo organization. The simplified nucleic acid model used short double-stranded DNA, so findings require validation with therapeutic-length RNA.

mRNA / LNP
Read guide →Original paper ↗

DOI: 10.1038/s41467-026-76810-y

Reviews & perspectives
Discover Nano

Critical chemistry manufacturing and controls considerations for mRNA lipid nanoparticle translation

Lanbo Liu; Yan Wang; Hua Gao; Jinghui Ruan; Wenna Lu; Tianzhu Liu; Xun Li; Guo-Qiang Zhang; Yating Xiao

This CMC review connects lipid chemistry and manufacturing parameters with critical quality attributes in mRNA-LNP development. It compares microfluidic scale-out with impinging-jet scale-up and emphasizes orthogonal analytics, stability in plasma or serum, lipid impurities, and RNA–lipid adducts. Early comparability planning is recommended. The framework does not demonstrate that one carrier or manufacturing platform is universally superior.

mRNA / LNP
Read guide →Original paper ↗

DOI: 10.1186/s11671-026-04864-4

Original research
Angewandte Chemie International Edition

Retinoic‐Acid‐Derived Ionizable Lipids Enable Spleen‐Selective Antigen Expression for mRNA Cancer Vaccination

Kai Jiang; Bing Shao; Runjie Li; Tianzhu Liang; Chengying You; Lili Wang; Jie Cen; Jiajia Tan; Yanhao Zhang; Minglong Chen; Shiyong Liu

A 40-member retinoic-acid-derived ionizable-lipid library yielded AT4-13CRA particles with preferential functional expression in splenic antigen-presenting cells. Researchers linked delivery to altered protein coronas and reduced lysosomal retention. An ovalbumin vaccination model showed stronger antigen-specific immunity and prophylactic tumor protection. The artificial antigen and challenge setting limit direct extrapolation to established human cancers.

mRNA / LNP
Read guide →Original paper ↗

DOI: 10.1002/anie.3823572

Original research
Nature Materials

An inherent T cell-activating mRNA delivery carrier for in vivo CAR T generation

Qiannan Cao; Yingli Yao; Wenming Zheng; Hongqian Liu; Mingxia Jiang; Dayang Xie; Siting Zhang; Pijun Su; Huilin Yuan; Xiaoyuan Chen; Huapan Fang; Huayu Tian

A polymer-lipid carrier incorporating arginine-modified oligoethylenimine combined T-cell activation with mRNA delivery without an added targeting ligand. The formulation favored splenic delivery and engaged PI3K-AKT-mTOR signaling. FAP-CAR mRNA generated functional CAR T cells in experimental cancer and fibrosis settings. These are preclinical results; the article also has a subsequent publisher correction that should accompany interpretation.

In Vivo Cell Engineering & Gene EditingmRNA / LNP
Read guide →Original paper ↗

DOI: 10.1038/s41563-026-02675-7

Original research
ACS Biomaterials Science & Engineering

Mannosylated Spleen-Targeted Lipid Nanoparticles Enable Enhanced mRNA Delivery to Antigen-Presenting Cells

Shu Zhang; Yaxi Li; Aygun Teymur; Bowen Yang; Sheereen Majd; Chandra Mohan; Tianfu Wu

A mannose-containing fifth lipid was incorporated into spleen-targeted LNPs to combine endogenous organ enrichment with receptor-associated uptake. Adjusting mannose-lipid content shifted distribution toward the spleen and improved macrophage transfection. Experiments identified uptake by macrophages and conventional dendritic cells, alongside freeze-thaw stability testing. These findings support immune-delivery development, while therapeutic and human targeting performance remain undetermined.

mRNA / LNP
Read guide →Original paper ↗

DOI: 10.1021/acsbiomaterials.6c00494

Original research
Nature Biomedical Engineering

Engineered lipid nanoparticles for in vivo and durable editing of haematopoietic stem cells within humanized mice

Jingjing Du; Zijin Luo; Dan Xie; Yan Chen; Ming Yang; Qiang Li; Lisha Wang; Lu Han; Yiming Zhang; Haiwei Li; Zilin Lan; Haoping Shi; Yinghui Li; Qiang Cheng; Fang Dong; Yingdai Gao; Yao Yao; Tao Cheng; Tuo Wei; Shuquan Rao

Anti-CD34-conjugated LNPs delivered reporter mRNA and gene-editing cargos to human hematopoietic stem and progenitor cells. Intrafemoral administration in humanized mice supported durable BCL11A-enhancer editing and fetal hemoglobin reactivation; ELANE editing partially restored neutrophil development in another model. The study addresses difficult stem-cell targeting but uses a specialized administration route and preclinical systems.

mRNA / LNP
Read guide →Original paper ↗

DOI: 10.1038/s41551-026-01765-w

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