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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
Nature

Cancer-selective frameshifting circuits show preclinical activity with systemic AAV9 delivery

Qiuyi Wang; Ao Chen; Yaoqi Liu; Xiaoxiao Yu; Hao Zhang; Yuqiang Yu; Pengyu Chen; Yue Zhang; Francis Ka-Ming Chan; Xiangwei Gao; Yuanhui Mao

A Nature study describes FAST gene circuits that exploit cancer-associated ribosomal frameshifting to conditionally express gasdermin D and trigger pyroptosis. According to the original abstract, systemic AAV9 delivery produced tumor regression and immune activation in mouse colorectal and pancreatic cancer models, with synergy alongside checkpoint blockade. These are cell and animal findings, not human clinical evidence.

In Vivo Cell Engineering & Gene Editing
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DOI: 10.1038/s41586-026-11200-4

Original research
Proceedings of the National Academy of Sciences

Bispecific protein drives dual receptor degradation in mouse eye-disease models

Jialun Zhang; Mingming Du; Lingying Ge; Yuheng Cao; Jiang Jiang; Xue Yin; Guangcun Chen; Qiangbin Wang

A PNAS study reports RHV, a tetravalent bispecific protein designed to internalize and lysosomally degrade VCAM-1 and VEGFR on pathological vessels. The original abstract describes activity in mouse choroidal neovascularization models, including efficacy comparable to aflibercept at one-quarter of the molar dose. This is preclinical extracellular receptor degradation, distinct from an intracellular small-molecule PROTAC.

Protein degradation / PROTAC
Read guide →Original paper ↗

DOI: 10.1073/pnas.2613631123

Original research
Nature Nanotechnology

Rational design of lipid nanoparticles for brain delivery of RNA therapeutics

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

Researchers used the properties of more than 100 central nervous system drugs to design brain-directed ionizable lipids. HX1 nanoparticles delivered RNA and enabled gene editing in mouse brains, delivered reporter mRNA in a nonhuman primate, and reduced radiation-related brain injury in mice carrying Il10 mRNA. These are preclinical findings.

mRNA / LNP
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DOI: 10.1038/s41565-026-02326-7

Reviews & perspectives
Frontiers in Chemistry

Review examines cocrystal performance and barriers to clinical translation

Muhammad Inam; Muhammad Hayat; Abuzar Osman

A Frontiers in Chemistry review examines pharmaceutical cocrystals as a way to modify dissolution and related biopharmaceutical properties without changing an API's chemical identity. The abstract distinguishes solubility-limited BCS II drugs from permeability-limited BCS IV drugs. It highlights gastrointestinal instability, manufacturing reproducibility, coformer selection, and limited pharmacokinetic evidence. Much of the reported performance evidence remains preclinical.

Pharmaceutical solid stateFormulation Development & Manufacturing
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DOI: 10.3389/fchem.2026.1965408

Reviews & perspectives
Bioanalysis

Review explains statistical pitfalls in anti-drug antibody assay cut points

Brijesh Kumar Verma

Based on the PubMed abstract, this review explains how sample selection, experimental balance, outlier handling, transformation, and statistical methods affect anti-drug antibody assay cut points. It addresses screening, confirmation, titers, drug tolerance, and reassessment using clinical baseline samples. This is a practical review of validation decisions, not a new clinical efficacy study.

Bioanalysis & Biomarkers
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DOI: 10.1080/17576180.2026.2758542

Methods
AAPS Open

Compaction density reveals reversible urea polymorph changes under pressure

Gerrit Vreeman; Changquan Calvin Sun

An AAPS Open study used a compaction simulator to track reversible density changes in urea between 600 and 900 MPa, consistent with its known form I-to-III transformation. The original abstract proposes in-die density monitoring as a material-sparing screen for pressure-induced phase changes. Applicability depends on a sufficiently large density difference; urea is the demonstrated model compound.

Pharmaceutical solid stateFormulation Development & Manufacturing
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DOI: 10.1186/s41120-026-00206-x

Reviews & perspectives
AAPS Open

Perspective maps oligonucleotide conjugation strategies beyond liver targeting

Ya Ying Zheng; Yonger Xue; Xucheng Hou; Yizhou Dong

An AAPS Open perspective surveys conjugation strategies for antisense oligonucleotides and siRNAs, including small-molecule ligands, cell-penetrating peptides, antibodies, and aptamers. The abstract contrasts clinically established GalNAc delivery with emerging approaches to extrahepatic tissues and discusses translation challenges. This is a synthesis of delivery platforms and clinical progress, rather than a new experimental or clinical efficacy study.

Oligonucleotide Therapeutics & Delivery
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DOI: 10.1186/s41120-026-00209-8

Reviews & perspectives
Bioanalysis

Review examines greener solvent systems for bioanalytical microextraction

Styliani Nisyriou; Marianna Ntorkou; Christina Patakidou; Constantinos K Zacharis

Based on the PubMed abstract, a Bioanalysis review examines switchable-hydrophilicity solvents and bio-derived deep eutectic solvents for sample preparation. It discusses extraction performance, matrix effects, reproducibility, and environmental trade-offs, with future emphasis on miniaturization and automation. The review evaluates methodological opportunities rather than validating a specific assay for regulated clinical sample analysis.

Bioanalysis & Biomarkers
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DOI: 10.1080/17576180.2026.2758030

Original research
Nature Communications

AI-driven discovery and engineering of human-derived protein scaffolds for mRNA delivery

Dongmin Xun; Lanqing Li; Jie Zou; Yi Liu; Ziyue Yang; Qianyi Liang; Ke Li; Zhu Zhu; Jicong Zhang; Lijun Lang; Kun Liu; Xinyu Wang; Meilin Tian; Guangyong Chen; Jun Yang; Bolin An; Pheng Ann Heng; Chao Zhong

The DeepDelivery platform screened the human proteome for proteins that could serve as mRNA delivery scaffolds. Twelve of 15 experimentally tested candidates supported particle formation and delivery within a modular system. Engineering TRIM25 domains improved performance. The work establishes a discovery framework in cultured cells, rather than clinical evidence of reduced immunogenicity.

mRNA / LNP
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DOI: 10.1038/s41467-026-79039-x

Methods
International Journal of Pharmaceutics

Simple heat-assisted method for mRNA loading into empty lipid nanoparticles enables robust in vitro and in vivo delivery

Takayuki Miyazaki; Kenji Kubara; Keita Kondo; Hiroshi Kamiyama; Shoki Hara; Jun Moriya; Hiroshi Ishihara; Yuta Suzuki

A post-loading method combined mRNA with preformed empty lipid nanoparticles using heat and acidic conditions, achieving roughly 90% encapsulation. The particles produced protein expression in mouse liver and spleen, although activity was slightly below conventional microfluidic formulations. Greater payload heterogeneity emerged as a potential limitation; the method supports 96-well preparation.

mRNA / LNP
Read guide →Original paper ↗

DOI: 10.1016/j.ijpharm.2026.127599

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

Overcoming technological and biological barriers to pulmonary mRNA delivery with lipid-polymer hybrid nanoparticles

Biesel A; Shandheep A; Pavlou A; Busker LM; Koch M; Kopliku V; Maisel K; Boese A; Skerra J; Schröder LJ; Neubert L; Schneider-Daum N; Loretz B; Kalinke U; Lehr CM

Lipid-polymer hybrid particles combined polymer stability with lipid-mediated transfection. Hyaluronic acid and polyvinyl alcohol coatings helped preserve particle properties after nebulization, while mucus-covered airway cultures retained measurable transfection. Primary human lung fibroblasts and intratracheally dosed mice provided additional delivery evidence. These results support aerosol formulation development, although intratracheal dosing does not establish the performance of inhaled aerosols in patients.

mRNA / LNP
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DOI: 10.1016/j.jconrel.2026.115509

Reviews & perspectives
Clinical Pharmacology & Therapeutics

Review identifies higher patient exposure for some drugs in chronic inflammation

Johannes Tillil; Sathej Gopalakrishnan; Jennifer Dong; Karthik Venkatakrishnan; Charlotte Kloft; Wilhelm Huisinga; Lena Klopp-Schulze

Based on the article abstract, a review of 20 approved small-molecule medicines for selected chronic inflammatory diseases found more than 25% higher exposure in patients for five medicines, using reported population pharmacokinetic analyses. The authors highlight CYP3A4 and CYP2C19 metabolism. This cross-drug review supports risk assessment, not automatic dose changes for individual patients.

Clinical Pharmacology & Model-Informed Development
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DOI: 10.1002/cpt.70535

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