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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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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
Read guide →Original paper ↗

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
Read guide →Original paper ↗

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
Read guide →Original paper ↗

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
Read guide →Original paper ↗

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
Read guide →Original paper ↗

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
Read guide →Original paper ↗

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
Read guide →Original paper ↗

DOI: 10.1039/d6ra07954g

Original research
Drug delivery and translational research

PEG-associated efficacy loss in lipid nanoparticles persists across formulation modifications for mRNA delivery

Mariah L. Arral; Namit Chaudhary; Katherine C. Fein; Alexandra N. Newby; Saigopalakrishna S. Yerneni; Jilian R. Melamed; Samuel T. LoPresti; Daria M. Strelkova Petersen; Kathryn A. Whitehead

This repeat-dosing study examined whether changes in PEG chemistry, PEG concentration, RNA cargo, or administration route could overcome efficacy loss after an immunogenic nanoparticle dose. None reliably restored delivery in the tested setting, and reduced activity tracked anti-PEG antibodies. The results emphasize the interaction between ionizable lipids and innate immunity, without establishing that every PEG-containing product will behave identically.

mRNA / LNP
Read guide →Original paper ↗

DOI: 10.1007/s13346-026-02225-3

Original research
Nature Communications

Interpretable AI-guided discovery of degradable ionizable lipids with enhanced hepatic mRNA delivery in vivo

Zhen Zhang; Yongbo Ren; Deqiang Kong; Jindong Feng; Kaiyi Hu; Xinyun Xu; Fukang Deng; Peixia Li; Jiuao Wang; Wenhui Huang; Guodan Wei; Yang Su; Ying Tan; Shuixing Zhang; Xiaodan Sun; Feiyu Kang; Hao Yan

Researchers combined a 520-member degradable lipid library with interpretable machine learning and a 10,920-member virtual design space. Independent mouse experiments identified PA97MD101 as a lead for hepatic mRNA expression, Cre-mediated recombination, and Cas9-based TTR suppression. The study links screening predictions with functional animal validation, without establishing clinical safety or efficacy.

mRNA / LNP
Read guide →Original paper ↗

DOI: 10.1038/s41467-026-78635-1

Original research
Science China. Life sciences

Optimization of lipid nanoparticles reduces hepatic accumulation and enhances local delivery of mRNA

Yue Ma; Yaoxian Han; Jiawei Zhou; Yuxin Xiao; Jie Liu; Wenjie Sun; Hui Yang; Guangyu Hu; Shangang Li; Weizhi Ji; Wenting Guo; Lei Zhang; Yongchang Chen

A design-of-experiments approach produced an SM-102-derived formulation with less hepatic reporter expression and preserved or improved expression after local administration. Experiments included intramuscular, intracerebroventricular, and intravitreal routes. Mechanistic analyses associated the change with altered protein binding, systemic exposure, and hepatocyte trafficking. These route-specific results support formulation optimization but do not demonstrate noninvasive access to the brain or eye.

mRNA / LNP
Read guide →Original paper ↗

DOI: 10.1007/s11427-025-3541-0

Original research
Journal of the American Chemical Society

Circular RNA Delivery In Vitro and In Vivo Using Doubly Branched Aminoethoxy-Functionalized Ionizable Lipid Nanoparticles

Adam Cook; Pavan K. Yadav; Ekram W. Helmy; Kseniia Vlasova; Howard Y. Chang; Paul A. Wender

A study designed ionizable lipids specifically for circular RNA, whose topology differs from linear mRNA. Lead lipid iL-19 combined aminoethoxy head groups with doubly branched tails and improved protein expression after intramuscular delivery in mice relative to several benchmark lipids. The results establish preclinical design principles, not clinical superiority.

mRNA / LNP
Read guide →Original paper ↗

DOI: 10.1021/jacs.6c14175

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

Lipid nanoparticle-mediated co-delivery of human RB1 mRNA and triptolide for localized genetic therapy of retinoblastoma

Chun K; Choi SY; An K; Jo DH; Lee H

A C12-200-based formulation was optimized for movement and cellular delivery in the protein-poor vitreous environment. It co-delivered RB1 mRNA and triptolide, improving antiproliferative activity and suppressing tumors in an orthotopic retinoblastoma model after intravitreal administration. Retinal evaluations supported tolerability during the study period. The combination remains preclinical and requires longer ocular safety assessment and pediatric translation studies.

mRNA / LNP
Read guide →Original paper ↗

DOI: 10.1016/j.jconrel.2026.115453

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