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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 136

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

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Original research
Nucleic Acid Therapeutics

GuNA-modified antisense gapmers show prolonged activity in mouse brain

Takaaki Kawanobe; Ajaya Ram Shrestha; Hideaki Tomita

Based on the PubMed abstract, researchers combined guanidine-bridged nucleic acid and MOE modifications in antisense gapmers, reporting improved knockdown in laboratory and animal experiments. Activity persisted beyond 24 weeks in mouse brain after intracerebroventricular administration. The study also examined immune responses and toxicity; these preclinical results do not establish human safety or efficacy.

Oligonucleotide Therapeutics & Delivery
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DOI: 10.1177/21593337261502560

Original research
Nature Biotechnology

An mRNA–lipid nanoparticle vaccine against H5N1 influenza virus protects lactating dairy cows from infection

Jefferson J. S. Santos; Carine K. Souza; Giovana C. Zanella; Débora B. Goulart; Bailey Arruda; Paola Boggiatto; Mitchell V. Palmer; Celeste A. Snyder; Michaela A. Kristula; Charlene Dickens; Terry L. Webb; Reilly K. Atkinson; Bernadeta Dadonaite; Garima Dwivedi; Mohamad-Gabriel Alameh; Christopher A. Hunter; Lisa A. Murphy; Jesse D. Bloom; Drew Weissman; Gary C. Althouse; Amy L. Baker; Scott E. Hensley

An experimental H5N1 mRNA-LNP vaccine was tested in eight lactating dairy cows split between vaccinated and control groups. After two doses and mammary challenge, vaccination reduced peak viral RNA in milk by about 1,000-fold and lessened mastitis and fibrosis. This small animal study does not establish field effectiveness or human protection.

mRNA / LNP
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DOI: 10.1038/s41587-026-03343-6

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

Uncovering the lymphatic uptake, pharmacokinetics, and biodistribution of mRNA-lipid nanoparticles after intravenous and subcutaneous injection

Peng Li; Mohammad Abdallah; Yuxin Yao; Rekha Shandre Mugan; Thomas J. Payne; Stewart A. Fabb; Colin W. Pouton; Natalie L. Trevaskis

Using three established ionizable lipids, researchers measured RNA, lipid components, lymphatic transport, plasma exposure, and tissue reporter expression in rats. After subcutaneous injection, lymph concentrations exceeded plasma concentrations, but lipid and RNA trafficking did not fully match. The study demonstrates why tracking a lipid label alone may not accurately represent intact RNA delivery or productive expression in tissues.

mRNA / LNP
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DOI: 10.1016/j.jconrel.2026.115491

Reviews & perspectives
Nature Reviews Materials

Programmable mRNA vaccine materials for temporal control of antitumour immunity

Seong Dong Jeong; Chaoyang Tang; Xiaotian Wang; Antoni J. Szeglowski; Yujin Kim; B. J. Kim; Padmanee Sharma; James J. Moon; Wen Jiang; Betty Y. S. Kim

A Perspective proposes designing mRNA vaccine materials around changing immune conditions before and after cancer surgery. The framework considers a broadly applicable preoperative prime followed by a personalized postoperative boost, with carriers controlling expression and immune activation. It is a conceptual design roadmap, not evidence from a new clinical trial.

mRNA / LNP
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DOI: 10.1038/s41578-026-00974-8

Original research
Molecular therapy : the journal of the American Society of Gene Therapy

A Scalable, Antibody-Free Lipid Nanoparticle Achieves Preferential mRNA Delivery to Human HSPCs In Vivo Without Conditioning

Kim E; Zenhausern R; Kapadia S; Johnson S; Cho S; Choi J; Park K; Diaz E; Podilapu AR; Kuppan R; Dahlman JE; Kim H; Woo J

A ligand-free nanoparticle formulation preferentially delivered mRNA to human hematopoietic stem and progenitor cells in humanized mice without conditioning. It reduced hepatic delivery relative to an SM-102 comparator and enabled functional Cas9-mediated editing of human CD34-positive cells. Single-cell analyses supported tolerability under the conditions examined. Durable stem-cell function and clinical delivery still require further investigation.

mRNA / LNP
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DOI: 10.1016/j.ymthe.2026.10.024

Original research
Molecular therapy : the journal of the American Society of Gene Therapy

A Combinatorial mRNA Therapy for Treating Rheumatoid Arthritis and Osteoarthritis by Inhibiting Inflammation and Preventing Cartilage Degeneration

Wang G; Guo Y; Guo K; Zhang C; Zhou Y; Xia J; Jiang H; Liu J; Ren J; Mohamed AO; Tang K; Chen X; Sun S; Xing Y; Yang Y; Shen M; Wang R; Huang Y; Li X; Lin A

An engineered nanoparticle delivered mRNAs encoding IL-1 receptor antagonist and an angiopoietin-like 3 derivative through intra-articular injection. The combination addressed inflammation and cartilage degeneration in several mouse arthritis models, with transcriptomic analyses supporting complementary biological effects. These findings broaden local mRNA therapy beyond vaccination, but durable structural benefit, repeated-dose safety, and effectiveness in human joints remain unproven.

mRNA / LNP
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DOI: 10.1016/j.ymthe.2026.10.021

Reviews & perspectives
Journal of Pharmaceutical Sciences

Particle occupancy in mRNA lipid nanoparticles: Measurement, manufacturing determinants, and relevance to product quality

Carlos Victor Montefusco-Pereira

A minireview distinguishes bulk encapsulation efficiency from particle occupancy and RNA copy-number distributions. High encapsulation efficiency can coexist with RNA-negative particles, and process changes may alter loading without changing average size. The author cautions that eliminating empty particles does not necessarily improve activity; product-specific evidence must establish whether occupancy is a critical quality attribute.

mRNA / LNP
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DOI: 10.1016/j.xphs.2026.104556

Original research
EMBO Molecular Medicine

Intramuscular self-amplifying RNA and adenoviral RSV vaccines program distinct systemic and respiratory mucosal immune architectures

Yao Zhang; Yu Qin; Zhongbing Qi; Qingzhe Yang; Xian Zhang; Shichuan Hu; Yuejun Li; Boduan Xiao; Jianchuan Hu; Ping Cheng

A mouse study compared LNP-delivered self-amplifying RNA and an Ad5 vaccine encoding the same RSV prefusion F antigen. Intramuscular dosing produced distinct immune patterns: saRNA favored pulmonary mucosal and tissue-resident memory responses, while Ad5 generated stronger systemic responses. The comparison concerns immune architecture, not demonstrated superiority in human protection.

mRNA / LNP
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DOI: 10.1038/s44321-026-00537-y

Original research
npj Vaccines

Preparation quality and epitranscriptomic modification influence circular RNA vaccine performance

Abby L. Martin; Jessica F. Cotterell; Kaitlin H. Buick; Thomas W. Bird; Joanna Kuang; Olga R. Palmer; Ngarangi C. Mason; Lydia White; Sarah L. Draper; Amy J. Foster; Gavin F. Painter; Isabelle Montgomerie; Tifany Oulavallickal; Wayne M. Patrick; Lisa M. Connor

Researchers separated the effects of circular structure, preparation quality, and nucleotide modification across two vaccine antigen systems. Cleaner circular RNA preparations caused less innate immune activation, but antibody and T-cell response levels were comparable with linear mRNA. Incorporating m6A was associated with higher antibody avidity, rather than a general increase in expression.

mRNA / LNP
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DOI: 10.1038/s41541-026-01581-y

Original research
International journal of pharmaceutics

Nebulization-stable lipid-polymer hybrid nanoparticles for mRNA delivery in advanced in vitro respiratory models

Hassoun H; Pascarel C; Erny A; Giang E; Garapon C; Gerossier L; Coiffier C; Monge C; Verrier B; Archer F; Aznar N

Researchers screened PEG-free hybrid particles containing a polylactic acid core and engineered lipid surface. A lead formulation preserved particle size and reporter activity after vibrating-mesh nebulization, outperforming the tested SM-102 reference under those conditions. Non-nebulized particles also transfected organoids and primary airway cultures. The authors identify mucus transport, aerosol deposition, and in vivo efficacy as unresolved next steps.

mRNA / LNP
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DOI: 10.1016/j.ijpharm.2026.127550

Methods
ACS Nano

In Vivo Blood Kinetics and Transcript Integrity of Three mRNA–Lipid Nanoparticle Vaccines in Humans

Stephen J. Kent; Shiyao Li; Thakshila H. Amarasena; Arnold Reynaldi; Michael G. Leeming; Jennifer A. Juno; Adam K. Wheatley; Georgia Deliyannis; Dale I. Godfrey; Terry Nolan; Colin W. Pouton; Miles P. Davenport; Yi Ju

Serial blood samples from 73 vaccine recipients were analyzed using ddPCR for total and long-range-linked mRNA and LC-MS for ionizable lipids. RNA and lipid decay differed across three vaccine platforms. Cohorts differed in dose, vaccination history, and sampling schedules. Detecting circulating RNA fragments does not by itself establish continued protein expression, clinical toxicity, or comparative vaccine effectiveness.

mRNA / LNP
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DOI: 10.1021/acsnano.6c09226

Original research
RSC Advances

40 years later: completion of the structural characterization of the anxiolytic drug buspirone HCl and solid-form landscape expansion

Delbert S. Botes; Mikaela I. Aleman; Alyssa M. Anderson; Pierre Le Maguerès; Kristin M. Hutchins

Electron diffraction established the previously unresolved structure of buspirone hydrochloride Form 2. The study also prepared 15 multicomponent solids, including cocrystals, an ionic cocrystal, and salts, and compared conformations and thermal behavior. This expands structural knowledge of a long-marketed drug and provides a solid-form exploration example without demonstrating improved clinical performance for the newly prepared materials.

MicroED structure determinationPharmaceutical solid state
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DOI: 10.1039/d6ra07954g

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