Every entry summarises what the study set out to test, what it found and why it changes how we design delivery systems. Browse research and reviews or search the collection.
586 articles
Showing 49–72 of 105 articles · Keyword: lipid nanoparticle Clear keyword
Zak Mm, Zangi L
mRNA therapeutics are limited by innate immune activation, rapid RNase degradation, and inefficient delivery to target organs. Although LNPs are the only clinically approved RNA therapeutic carriers, most systemically delivered LNPs accumulate in the liver, so organ-specific delivery remains a major barrier for…
Read the articleRumiana Tenchov, Robert E. Bird, Allison E. Curtze, Qiongqiong Angela Zhou
Lipid nanoparticles (LNPs) have become key delivery vehicles across pharmaceuticals, with renewed prominence as the delivery platform for COVID-19 mRNA vaccines. The field lacks a broad, quantitative landscape of LNP research across liposomes, solid lipid nanoparticles, nanostructured lipid carriers, cationic…
Read the articleAshok B, Peppas Na, Wechsler Me
CRISPR/Cas9 genome editing holds major therapeutic promise, but in vivo delivery is arguably the most difficult barrier to clinical translation. Efficient, selective transport of editing components to target cells/tissues while minimising off-target effects remains unresolved. This review critiques lipid- and…
Read the articleRui Zhang, Rakan El-Mayta, Timothy Murdoch, Claude Warzecha, Margaret Billingsley, Sarah Shepherd, Ningqiang Gong, Lili Wang, James Wilson, Daeyeon Lee, Michael J. Mitchell
LNP delivery to liver versus spleen is difficult to predict from formulation parameters, and the role of helper phospholipids in biodistribution and protein adsorption is incompletely understood. This study probed how helper lipid identity (DOPE vs DSPC) affects LNP accumulation in liver/spleen and interactions with…
Read the articleYusuke Sato, Nana Okabe, Yusuke Note, Kazuki Hashiba, Masatoshi Maeki, Manabu Tokeshi, Hideyoshi Harashima
Small-sized lipid nanoparticles (LNPs) are attractive for tissue penetration but often lose potency because lipid components diffuse out and serum proteins adsorb onto poorly packed surfaces. The study asked how the hydrophobic scaffold structure of pH-sensitive cationic lipids affects small-LNP stability, lipid…
Read the articleXuewei Zhao, Jinjin Chen, Min Qiu, Yamin Li, Zachary Glass, Qiaobing Xu
In vivo engineering of T lymphocytes is limited by poor transfection efficiency and reliance on ex vivo electroporation. A non-viral mRNA delivery platform that can target primary T cells in vivo is needed for CAR T-cell therapy, vaccines, and immunotherapy. Rough screening identified imidazole head 93 with O17O…
Read the articleMargaret M. Billingsley, Nathan Singh, Pranali Ravikumar, Rui Zhang, Carl H. June, Michael J. Mitchell
CAR T cell therapy currently relies on viral vectors or electroporation for T cell engineering. Viral vectors cause permanent CAR expression and carry safety/manufacturing limitations, while electroporation is cytotoxic and requires specialised equipment. A safer, efficient non-viral mRNA delivery platform for human…
Read the articlePedro P. Guimaraes, Rui Zhang, Roman Spektor, Mingchee Tan, Amanda Chung, Margaret M. Billingsley, Rakan El-Mayta, Rachel S. Riley, Lili Wang, James M. Wilson, Michael J. Mitchell
LNP libraries are usually screened in vitro, which poorly predicts in vivo mRNA delivery. A high-throughput in vivo platform was needed to screen many LNP formulations simultaneously and identify lead carriers for functional mRNA delivery. b-mRNA could be detected in liver at doses as low as 17 ng; b-mRNA delivery…
Read the articleNour Shobaki, Yusuke Sato, Yuichi Suzuki, Nana Okabe, Hideyoshi Harashima
Tumour-associated macrophages (TAMs) are largely M2-like and promote tumour growth, angiogenesis, metastasis, and immune suppression. There is a need for systemic delivery systems that can target TAMs and manipulate their pro-tumour functions with siRNA for cancer immunotherapy. Optimised CL4H6-LNPs had 90% siRNA…
Read the articleSiddharth Patel, N. Ashwanikumar, Ema Robinson, Yan Xia, Cosmin Mihai, Joseph P. Griffith Iii, Shangguo Hou, Adam A. Esposito, Tatiana Ketova, Kevin Welsher, John L. Joyal, Örn Almarsson, Gaurav Sahay
Endosomal sequestration of lipid-based nanoparticles (LNPs) remains a formidable barrier to delivery, with only <2% of LNPs reaching the cytosol. The role of cholesterol structure in LNP-mediated mRNA delivery and endosomal escape was poorly understood. There is a need to decode the structural characteristics of…
Read the articleZeb A, Rana I, Choi H-I, Et Al.
Biopharmaceuticals have become clinically and commercially important due to their specificity, potency, targeting ability, and reduced side effects, but their macromolecular structure and intrinsic instability make formulation and administration challenging; parenteral delivery remains the only viable option in most…
Read the articleKaren Bulaklak And Charles A. Gersbach
Gene therapy is at an inflection point: recent approvals and clinical successes have validated the field, but broader application is still limited by immune responses to vectors, inefficient delivery outside the retina and liver, and regulatory frameworks that are poorly suited to personalised, potentially curative…
Read the articleGuevara Ml, Persano F, Persano S
mRNA is a flexible and potentially safer cancer immunotherapy platform, but its clinical use has been limited by extracellular instability, poor cellular uptake, and inefficient endosomal escape. Lipid nanoparticles have become the most advanced non-viral delivery system for mRNA, enabling therapeutic vaccines…
Read the articleFrancia V, Schiffelers Rm, Cullis Pr, Witzigmann D
LNPs are clinically advanced vectors for siRNA, mRNA, and DNA delivery, but their in vivo behaviour is strongly influenced by the biological environment. Upon administration, biomolecules adsorb to the LNP surface and form a “biomolecular corona,” which defines the particle’s biological identity, affects stability…
Read the articleLiu C, Zhang L, Dong N, Zhang W, Chen X, Gao R, Sun H.
Cationic liposomes (CLs) are promising non-viral gene-delivery vectors, but their clinical use in cancer gene therapy is limited by extracellular barriers (opsonisation, RES clearance, poor tumour penetration) and intracellular barriers (endosomal/lysosomal entrapment, restricted cytoplasmic/nuclear transport). The…
Read the articleWahane A, Waghmode A, Kapphahn A, Dhur K, Gupta A, Bahal R
Nucleic acid analogs and genome-editing proteins have broad therapeutic potential, but clinical translation is limited by poor cell- and organ-specific delivery, endosomal entrapment, enzymatic degradation, and short bioavailability. The review examines lipid- and polymer-based non-viral vectors as strategies to…
Read the articlePiotr S. Kowalski, Arnab Rudra, Lei Miao, Daniel G. Anderson
mRNA has broad therapeutic potential for vaccination, protein replacement, and gene editing, but its clinical translation is limited by the need for improved intracellular delivery systems. mRNA is much larger than siRNA or ASOs (~300–5,000 kDa, ~1–15 kb vs ~14 kDa and 4–10 kDa, respectively), making delivery more…
Read the articleDongyu Chen, Neha Parayath, Shanthi Ganesh, Weimin Wang, Mansoor Amiji
The protein corona that forms on lipid nanoparticles (LNPs) after systemic administration can alter their biological identity, but its impact on tumour delivery is not well understood. This study aimed to determine whether LNP formulation composition and surface charge can manipulate the protein corona and thereby…
Dongyu Chen, Shanthi Ganesh, Weimin Wang, Mansoor Amiji
Protein corona formation in biological fluids can alter nanoparticle cellular uptake, transfection, and gene silencing, but the relationship between LNP surface chemistry and corona-mediated delivery is incompletely understood. This study examined how PEG lipid chain length and PEG molar ratio affect protein corona…
Read the articleYusuke Sato, Kazuki Hashiba, Kosuke Sasaki, Masatoshi Maeki, Manabu Tokeshi, Hideyoshi Harashima
Endosomal escape remains a major rate-limiting step for siRNA delivery. The structure–activity relationships (SAR) of pH-sensitive cationic lipids were incompletely understood, limiting rational design of lipid nanoparticles (LNPs) for efficient in vivo siRNA delivery to hepatocytes. Hydrophilic head group structure…
Read the articleYuhang Jiang, Qiao Lu, Yongheng Wang, Emily Xu, Alison Ho, Priya Singh, Yifei Wang, Zhaozhong Jiang, Fan Yang, Gregory T. Tietjen, Peter Cresswell, W. Mark Saltzman
Endosomal escape is a key but poorly quantified step for intracellular nucleic acid delivery. Existing methods are low-throughput, indirect, or fail to distinguish endosomal disruption from actual cytosolic cargo delivery. A reliable, sensitive method is needed to rationally optimise polymeric mRNA delivery vehicles…
Read the articleMukalel Aj, Riley Rs, Zhang R, Mitchell Mj.
Nucleic acid therapeutics—DNA, mRNA, and CRISPR/Cas9 genome editing systems—hold significant potential for cancer immunotherapy but face major delivery barriers, including rapid in vivo degradation, poor cellular uptake, required nuclear entry, and toxicity in healthy tissues. Nanoparticle delivery systems are being…
Read the articleZhao W, Hou X, Vick Og, Dong Y.
Genetic and rare diseases (GARDs) affect more than 350 million patients worldwide, and many lack effective treatments. RNA-based therapeutics—siRNA, mRNA, miRNA, lncRNA, and RNA-containing genome editing systems—are promising, but naked RNA has poor cellular penetration, is degraded in biological fluids, and does not…
Read the articleDhrisya Chenthamara, Sadhasivam Subramaniam, Sankar Ganesh Ramakrishnan, Swaminathan Krishnaswamy, Musthafa Mohamed Essa, Feng-Huei Lin, And M. Walid Qoronfleh
Despite enormous strides in nanotechnology research and development, it is often confusing for beginners to make an informed choice regarding the nanocarrier system and its potential applications. There is a need to provide a comprehensive overview of commonly used nanomaterials, their core properties, surface…
Read the articleWe co-develop nanocarrier and biosensing programmes with pharma, biotech and academic groups — from target selection through scale-up.
