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Papers, explained in our own words

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.

706 articles

Showing 73–96 of 120 articles · Keyword: siRNA Clear keyword

2017ReviewNon-viral Gene Delivery

73. Liposomal Delivery Systems: Design Optimization and Current Applications

Liposomes are attractive pharmaceutical carriers because they encapsulate hydrophilic and hydrophobic drugs and are biocompatible and biodegradable. However, conventional liposomes suffer from aggregation/fusion, premature payload release, rapid clearance by the mononuclear phagocyte system (MPS), low entrapment…

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2017ReviewDrug Delivery

74. Liposomal Formulations in Clinical Use: An Updated Review

Liposomes are the first nano drug delivery systems successfully translated into clinical use, but many formulations still face limitations in biodistribution, toxicity, stability, and target selectivity. An updated review was needed to summarise liposome technologies, marketed clinical products, formulation aspects…

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International Journal of Pharmaceutics.2017ReviewNon-viral Gene Delivery

75. Targeted si-RNA with liposomes and exosomes (extracellular vesicles): How to unlock the potential

RNA interference (RNAi) therapeutics have shown potential for treating genetic, viral, and cancerous diseases, but the main bottleneck remains delivery of functional RNA molecules into the cell cytoplasm. The review focuses on liposomes and other lipid carriers, and on exosomes/extracellular vesicles (EVs), as…

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WIREs Nanomedicine and Nanobiotechnology2017ReviewNon-viral Gene Delivery

76. Nanoescapology progress toward understanding the endosomal escape of polymeric nanoparticles

Selby, L. I.; Cortez-Jugo, C. M.; Such, G. K.; Johnston, A. P. R

Endosomal escape is widely considered the rate-limiting bottleneck for cytoplasmic delivery of nucleic acids, proteins, and other therapeutics using polymeric and lipid nanoparticles. The field lacks robust methods to quantify escape efficiency and understand its mechanisms, limiting rational design of improved…

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Journal of Controlled Release2016ReviewNon-viral Gene Delivery

77. Materials for Non-Viral Intracellular Delivery of Messenger RNA Therapeutics

Kevin J. Kauffman, Matthew J. Webber, Daniel G. Anderson

mRNA therapeutics have broad potential for protein replacement therapy, cancer immunotherapy, and genomic engineering, but effective intracellular delivery remains a major challenge. A chemically diverse suite of delivery materials—originally developed for DNA and siRNA transfection—has recently shown promise for…

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Materials Science and Engineering C2016ResearchNon-viral Gene Delivery

78. Gene delivery efficiency and cytotoxicity of heterocyclic amine-modified PAMAM and PPI dendrimers

Maryam Hashemi, Seyed Meghdad Tabatabai, Hamideh Parhiz, Soroush Milanizadeh, Sara Amel Farzad, Khalil Abnous, Mohammad Ramezani

PAMAM and PPI dendrimers are widely investigated for gene delivery but suffer from limitations including cytotoxicity and inefficient endosomal escape. While both employ the proton sponge mechanism for endosomal release, higher generations that improve transfection also increase cytotoxicity. A systematic comparison…

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Journal of Controlled Release2016ResearchNon-viral Gene Delivery

79. Development of siRNA-loaded chitosan nanoparticles targeting Galectin-1 for the treatment of glioblastoma multiforme via intranasal administration

Matthias Van Woensel, Nathalie Wauthoz, Rémi Rosière, Véronique Mathieu, Róbert Kiss, Florence Lefranc, Brecht Steelant, Ellen Dilissen, Stefaan W. Van Gool, Thomas Mathivet, Holger Gerhardt, Karim Amighi, Steven De Vleeschouwer

Galectin-1 (Gal-1) is overexpressed in glioblastoma multiforme (GBM), promotes tumour progression, angiogenesis, chemoresistance, and immune suppression. A non-invasive delivery strategy is needed to reach CNS tumours with siRNA while limiting systemic effects. The intranasal route was explored as an underexplored…

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Molecular Therapy — Methods & Clinical Development2016ReviewNon-viral Gene Delivery

80. Production and clinical development of nanoparticles for gene delivery

Chen J, Guo Z, Tian H, Chen X.

Gene therapy is a promising strategy for treating gene-associated diseases, but clinical application is limited by inefficient delivery. Naked nucleic acids suffer from nuclease degradation, rapid renal clearance, phagocyte uptake, poor target-cell uptake, and immune-related toxicity. Nanoparticles are considered the…

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Advanced Drug Delivery Reviews.2016ReviewNon-viral Gene Delivery

81. The role of helper lipids in lipid nanoparticles (LNPs) designed for oligonucleotide delivery

Cheng X, Lee Rj

Oligonucleotide (ON) therapeutics—antisense ONs, siRNA, miRNA mimics/inhibitors, antimiRs—are limited by poor membrane permeability, rapid renal/RES clearance, and nuclease degradation. LNPs can protect ONs and improve delivery, but the role of helper lipids in LNP stability, circulation, endosomal escape, and in…

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Cell Chemical Biology (Chemistry & Biology)2015ResearchNon-viral Gene Delivery

82. Attachment of Cell-Binding Ligands to Arginine-Rich Cell-Penetrating Peptides Enables Cytosolic Translocation of Complexed siRNA

Skye Zeller, Chang Seon Choi, Pradeep D. Uchil, Hong-Seok Ban, Alyssa Siefert, Tarek M. Fahmy, Walther Mothes, Sang-Kyung Lee, Priti Kumar

Arginine-rich cell-penetrating peptides (CPPs) such as nona-arginine (9R) can effectively translocate covalently attached biomolecules into cells, but poorly deliver electrostatically complexed siRNA, with most CPP:siRNA complexes trapped in endosomes. The mechanism by which ligand attachment to 9R overcomes this…

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2015ReviewNon-viral Gene Delivery

83. Lipid-Based Nanocarriers for RNA Delivery

Xue Hy, Guo P, Wen W-C, Wong Hl

RNAi therapeutics such as siRNA and miRNA can potently and specifically silence almost any gene, but systemic delivery is limited by poor cell permeation, rapid nuclease degradation, immunogenicity, and lack of target specificity. Lipid-based nanocarriers are among the most biocompatible and clinically advanced…

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Journal of Controlled Release.2015ReviewNon-viral Gene Delivery

84. Solid lipid nanoparticles as nucleic acid delivery system Properties and molecular mechanisms

De Jesus Mb, Zuhorn Is

Non-viral nucleic acid delivery requires carriers that protect DNA/RNA from nucleases and facilitate intracellular delivery. Solid lipid nanoparticles (SLNs) are promising, but the molecular mechanisms by which they assemble with nucleic acids, transfect cells, and release cargo remain poorly understood compared with…

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Journal of Controlled Release2015ReviewDrug Delivery

85. Amphiphilic poly(amino acid) based micelles applied to drug delivery The in vitro and in vivo challenges and the corresponding potential strategies

Helin Xu, Qing Yao, Cuifang Cai, Jinxin Gou, Yu Zhang, Haijun Zhong, Xing Tang

Amphiphilic poly(amino acid)-based micelles are attractive for systemic drug delivery because they are biodegradable, biocompatible, have functional side groups, can load diverse drugs, and can exploit the EPR effect. However, low cargo capacity and poor stability in blood remain major barriers to clinical…

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Chemical Reviews2015ReviewNon-viral Gene Delivery

86. Biodegradable Polymer Nanogels for DrugNucleic Acid Delivery

Li, Y.; Maciel, D.; Rodrigues, J.; Shi, X.; Tomás, H

Despite extensive research, therapeutic delivery still faces major barriers: poor plasma stability, rapid clearance by the reticuloendothelial system (RES), and intracellular obstacles such as endosomal entrapment and lysosomal degradation. Biodegradable polymer nanogels (NGs) — physically or chemically cross-linked…

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Journal of Controlled Release2015ReviewNon-viral Gene Delivery

87. Exploring the role of polymer structure on intracellular nucleic acid delivery via polymeric nanoparticles

Bishop, C. J.; Kozielski, K. L.; Green, J. J

Viral vectors raise safety concerns, and non-viral polymeric vectors still require optimisation for clinical translation. This review addresses how polymer structural features govern intracellular nucleic acid delivery — including cargo protection, cellular uptake, endosomal escape, unpacking, and nuclear delivery —…

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Journal of drug targeting2015ReviewNon-viral Gene Delivery

88. Targeted polymeric nanoparticles for cancer gene therapy

Kim J, Wilson Dr, Zamboni Cg, Green Jj

Cancer gene therapy requires safe and efficient delivery of nucleic acids, but viral vectors carry risks of immunogenicity and insertional tumorigenicity, while non-viral polymeric vectors are safer but less efficient. The review addresses the need for targeted polymeric nanoparticles that achieve tumour-specific…

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Chemical Reviews2015ReviewNon-viral Gene Delivery

89. Nucleic Acid Therapeutics Using Polyplexes: A Journey of 50 Years (and Beyond)

Ulrich Lächelt And Ernst Wagner

Therapeutic nucleic acids—including plasmid DNA, mRNA, siRNA, miRNA, antisense oligonucleotides, and aptamers—have broad therapeutic potential, but their delivery to the correct intracellular site of action remains the dominant bottleneck. Polyplexes, formed by electrostatic complexation of nucleic acids with…

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Cold Spring Harbor Perspectives in Biology2014ReviewNon-viral Gene Delivery

90. Clathrin-Independent Pathways of Endocytosis

Satyajit Mayor, Robert G. Parton, Julie G. Donaldson

Clathrin-mediated endocytosis (CME) has long been the dominant paradigm for cellular uptake, but many endocytic pathways operate without clathrin. These clathrin-independent endocytic (CIE) pathways are increasingly recognised as distinct routes with different cargo selectivity, molecular machinery, kinetics, and…

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Nanoscale2014ReviewNon-viral Gene Delivery

91. Enhancing Endosomal Escape for Nanoparticle Mediated siRNA Delivery

Da Ma

siRNA-based gene therapy is a promising biotechnology for treating cancer and other diseases, but safe and efficient delivery is essential. Nanoparticle-mediated delivery must overcome multiple biological barriers, and endosomal escape is a critical rate-limiting barrier: nanoparticles are typically internalised via…

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Acta Biomaterialia2014ResearchNon-viral Gene Delivery

92. Degradable Polymer-Coated Gold Nanoparticles for Co-Delivery of DNA and siRNA

Corey J. Bishop, Stephany Y. Tzeng, Jordan J. Green

There is a need for improved nanobiotechnologies that enable intracellular delivery of difficult-to-deliver biologics such as nucleic acids. Ideally, a delivery material should deliver both large molecules such as DNA and small molecules such as siRNA—enabling both positive and negative regulation of genes—while…

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Journal of Controlled Release2014ResearchNon-viral Gene Delivery

93. Chitosan nanoparticles for siRNA delivery Optimizing formulation to increase stability and efficiency

Héloïse Ragelle, Raphaël Riva, Gaëlle Vandermeulen, Broes Naeye, Vincent Pourcelle, Cécile S. Le Duff, Cécile D’haese, Bernard Nysten, Kevin Braeckmans, Stefaan C. De Smedt, Christine Jérôme, Véronique Préat

Naked siRNA is unstable and inefficient, and chitosan-based carriers are limited by poor solubility at physiological pH and weak endosomal disruption. The study aimed to optimise chitosan nanoparticle formulations for intravenous siRNA delivery that achieve high gene silencing without cytotoxicity and remain stable…

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Nature Reviews Genetics2014ReviewNon-viral Gene Delivery

94. Non-viral vectors for gene-based therapy

Yin H, Kanasty Rl, Eltoukhy Aa, Vegas Aj, Dorkin Jr, Anderson Dg.

Gene-based therapy has potential to treat a wide range of diseases, but clinical success is limited by delivery barriers. Viral vectors, while efficient, carry risks of carcinogenesis, immunogenicity, broad tropism, limited packaging capacity, and manufacturing difficulties. Non-viral vectors offer improved safety…

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BioMed Research International.2014ReviewNon-viral Gene Delivery

95. Lipid Nanoparticles as Carriers for RNAi against Viral Infections Current Status and Future Perspectives

Torrecilla J, Rodríguez-Gascón A, Solinís Má, Del Pozo-Rodríguez A

Naked RNAi molecules are rapidly degraded by serum nucleases and cannot readily cross cell membranes because of their negative charge. Effective delivery systems are therefore required to exploit RNAi for antiviral therapy. Lipid nanoparticles (LNPs) are attractive because they are relatively safe, simple to produce…

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European Journal of Pharmaceutics and Biopharmaceutics.2014ReviewNon-viral Gene Delivery

96. Nanotoxicology applied to solid lipid nanoparticles and nanostructured lipid carriers – A systematic review of in vitro data

Slavomíra Doktorovová, Eliana B. Souto, Amélia Maria Silva

Solid lipid nanoparticles (SLN) and nanostructured lipid carriers (NLC) were developed as alternative colloidal carriers with better stability and tolerability than polymeric nanoparticles. However, in vitro toxicity data were scattered across many cell lines, assays, exposure times, concentrations, and formulations…

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