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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.

Original research
Journal of virology

A multivalent mRNA-lipid nanoparticle vaccine containing eight hemagglutinin antigens elicited broad neutralizing antibody responses and protected against influenza A virus challenge in swine

Ciacci Zanella G; Vincent ML; Flores L; Aljets EK; Paiva RC; Markin A; Inderski B; Dwivedi G; Weissman D; Wymore Brand M; Santos JJS; Hensley SE; Anderson TK; Gauger PC; Baker AL

Researchers selected eight hemagglutinin sequences to represent circulating swine influenza diversity and evaluated individual and multivalent mRNA vaccines in pigs. The multivalent formulation elicited responses against its component antigens and protected against lung lesions after the tested challenge. This large-animal veterinary study supports adaptable multivalent design, while protection across untested strains and direct relevance to human influenza remain unresolved.

DOI: 10.1128/jvi.00848-26

Original research
Vaccine

Circular RNA vaccines encoding Pvs25 induce antibodies that block Plasmodium vivax transmission

Nawapol Kunkeaw; Wang Nguitragool; Thitipa Thosapornvichai; Nattawut Buasri; Suradej Hongeng; Eizo Takashima; Takafumi Tsuboi; Patompon Wongtrakoongate; Jetsumon Sattabongkot

Circular RNA nanoparticles encoding the malaria transmission antigen Pvs25 were compared with a modified linear mRNA vaccine in mice. Both platforms induced antigen-specific responses, and booster immunization generated antibodies that blocked parasite development in mosquito assays. The findings support further evaluation of circular RNA for transmission-blocking vaccination, without establishing greater efficacy, durability, or manufacturing economy than linear mRNA in humans.

DOI: 10.1016/j.vaccine.2026.129221

Original research
iScience

Lipid-based mRNA chimeric antigen receptor T cells for enhanced <i>in vitro</i> antitumor activity

Haixia Gao; Yuan Fang; Zhidan Tian; Chuanxin Guo; Pei Wang; Tao Liu; Yan Sun; Pingjing Zhang; Qijun Qian

Screening eight ionizable lipids identified a lead formulation that delivered reporter RNA to several immune-cell types. Researchers then used it to engineer primary human T cells expressing a MUC1-directed CAR, with cytotoxic activity against antigen-positive tumor cells in vitro. Animal reporter experiments supported delivery feasibility, but the study does not demonstrate therapeutic CAR-T generation inside patients or animals.

DOI: 10.1016/j.isci.2026.117390

Original research
Molecular Pharmaceutics

Formulations at the Nanoscale: Multi-dimensional Electron Diffraction of Long-Acting Injectable, Cabotegravir

Helen W. Leung; Royston C. B. Copley; Stephanie Lam; Luca Russo; Simon Fairclough; Duncan N. Johnstone; Paul A. Midgley

This study applied multidimensional electron diffraction to cabotegravir nanocrystals within a long-acting injectable formulation. 3D electron diffraction confirmed a structure consistent with the known X-ray model, while imaging and diffraction linked particle shape to crystal structure and revealed dislocations. The results describe nanoscale structural features that may guide formulation work; improved product performance was not demonstrated.

DOI: 10.1021/acs.molpharmaceut.6c00365

Original research
Journal of Medicinal Chemistry

Discovery of a Quinazoline-Based Nav1.7 Inhibitor with MicroED-Confirmed Absolute Configuration and Oral Efficacy in Rodent Models of Neuropathic Pain

Jae Ho Shim; Pooreum Lim; Sangwoo Woo; Jihye Han; Hyeon Soo Kim

A medicinal-chemistry study developed a chiral quinazoline-based Nav1.7 inhibitor and used MicroED to confirm the lead compound’s absolute configuration. The report combined structure–activity studies with pharmacokinetic and pain-model experiments in rodents. Its pharmaceutical relevance is the stereochemical assignment within a drug-discovery program; the animal findings do not establish analgesic efficacy or safety in patients.

DOI: 10.1021/acs.jmedchem.6c02422

Reviews & perspectives
Discover Pharmaceutical Sciences

A review of analytical techniques for characterization of mRNA drug substance in therapeutics and their suitability as a multi-attribute method

Kyle J. Tynan; Karndeep Singh; Trina Mouchahoir; Mark S. Lowenthal; Karen W. Phinney

This review evaluates electrophoresis, chromatography, sequencing, and mass spectrometry for measuring mRNA drug-substance identity, length, concentration, intactness, and purity. It compares throughput, sample preparation, and total analysis time while considering multi-attribute methods used for protein therapeutics. The authors identify opportunities for integrated mRNA analytics, rather than validating a universal platform that replaces all individual quality assays.

DOI: 10.1007/s44395-026-00058-0

Original research
Journal of controlled release : official journal of the Controlled Release Society

Protein expression kinetics of mRNA, saRNA, and pDNA as individual or co-delivered payloads using a common lipid nanoparticle formulation

McMillan C; Hussain M; Punnabhum P; Varley AJ; Kong KYS; Rattray Z; Druschitz A; Perrie Y

Using a common ALC-0315 formulation, the study compared reporter expression from conventional mRNA, self-amplifying RNA, and plasmid DNA, individually and in combinations. Mouse experiments showed distinct timing and duration associated with payload identity. Mixed preparations generally behaved additively under the tested conditions. The work helps separate cargo effects from formulation effects, without establishing universal expression profiles across doses, tissues, or products.

DOI: 10.1016/j.jconrel.2026.115418

Original research
ACS nano

Targeted Gastrointestinal Delivery of mRNA Lipid Nanoparticles Enables Systemic Vaccination and Glucagon-like Peptide-1 Metabolic Therapy

Abdulraouf M. Abbas; Ramy Ghanim; Sebastian Rudden; David Schultz; Bora Jang; Avraham Shakked; Hyejin Kim; James E. Dahlman; Alex Abramson

Investigators administered nanoparticles to several gastrointestinal submucosal sites to examine route-dependent targeting. Gastric delivery increased pancreatic exposure in rodents, while GLP-1 mRNA improved glucose regulation in mice. Buccal administration supported local and lymphatic targeting with vaccine responses. These are tissue-directed administration experiments; they do not establish that conventional swallowed nanoparticles can survive digestion and reproduce the same delivery profile.

DOI: 10.1021/acsnano.5c18549

Original research
Nature biotechnology

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; Xianglei Fu; Sijin Luozhong; Muye Zhou; Payton Kirtley; Brandon K. Wilder; Bowen Li

A lipid screen explicitly incorporated a large base-editor transcript rather than relying on small reporter RNA. The lead lipid retained favorable RNA interactions and membrane-disrupting structure as cargo size increased. It improved editing in liver, brain, and lung using different administration routes, including intrathecal and intratracheal dosing. The findings do not establish systemic delivery to all three organs.

DOI: 10.1038/s41587-026-03298-8

Methods
Nature Biotechnology

Accelerated discovery of thermostable mRNA–lipid nanoparticle vaccines using data-efficient AI

Jinbi Tian; Khanh T. M. Tran; Brett H. Pogostin; Olivia Sheridan; Sevinj Mursalova; Amy H. Lee; Shuai Liu; Jaya Hamkins; Daniel Antov; Alana L. Power; Zane S. Dash; Dongsoo Yun; Mina Konaković Luković; Robert S. Langer; Ana Jaklenec

The AGENT platform combines Bayesian optimization with high-throughput experiments to identify thermostable mRNA-LNP formulations through six iterations in approximately one month. Solid-state model formulations based on SM-102 and ALC-0315 retained biological activity after more than two months at 37°C. Animal immunogenicity supported further development, but these formulation-specific findings do not eliminate cold-chain requirements for mRNA products generally.

DOI: 10.1038/s41587-026-03331-w

Methods
Advanced Materials

Increasing Lipid Nanoparticle mRNA Copy Number Improves Transfection Potency

Yao Zhang; Jerry Leung; Judy Cheng; Arash Momeni; Albert Kamanzi; Ariadne Tuckmantel Bido; Suiyang Liao; Emma Kang; Colton Strong; Christian J. Kastrup; Sabrina Leslie; Miffy Hok Yan Cheng; Pieter R. Cullis

Researchers modified buffer exchange into a two-step dialysis process, first promoting fusion under acidic, higher-ionic-strength conditions while retaining ethanol, then removing ethanol and raising pH. Compared with one-step exchange, the process increased mRNA copies per particle, reduced empty particles, and improved transfection in cells and animals. These formulation-specific results do not establish that removing empties always improves every product.

DOI: 10.1002/adma.75174

Original research
Advanced science (Weinheim, Baden-Wurttemberg, Germany)

Immune Checkpoint Inhibitor as a Fifth Component of Lipid Nanoparticles Enables Lymph Node-Targeting and Augments mRNA Vaccine Efficacy

Ha M; Kim J; Kim S; Naidu GS; Cho S; Yong SB

Researchers incorporated an amphiphilic PD-L1 inhibitor as an additional nanoparticle component rather than administering it separately. The modified formulation increased lymph-node reporter expression and strengthened vaccine-associated immune responses in experimental models. Similar targeting effects were observed with two ionizable lipid backbones. These results suggest formulation-dependent immune targeting, without proving broad applicability or clinical benefit across tumor types.

DOI: 10.1002/advs.77923

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