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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 167 articles · Keyword: DNA Clear keyword

Molecular Therapy2017ReviewNon-viral Gene Delivery

73. Lipid Nanoparticle Systems for Enabling Gene Therapies

Cullis Pr, Hope Mj

Genetic drugs such as siRNA, mRNA, and plasmid DNA could treat most diseases by silencing pathological genes, expressing therapeutic proteins, or enabling gene editing. However, naked RNA/DNA is rapidly degraded, does not accumulate in target tissues, and cannot cross cell membranes efficiently. Lipid nanoparticles…

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Gene Therapy2017ReviewNon-viral Gene Delivery

74. Engineering liposomal nanoparticles for targeted gene therapy

Zylberberg C, Gaskill K, Pasley S, Matosevic S

Liposome-mediated gene delivery faces systemic barriers, endosomal escape, lysosomal degradation, and nuclear uptake limitations, while interactions between lipid nanoparticles and cells remain poorly understood. Rational design of targeted liposomes is needed to reduce off-target effects and enhance transfection…

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Journal of Controlled Release2016ReviewNon-viral Gene Delivery

75. 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

76. 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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Nanomedicine: Nanotechnology, Biology, and Medicine2016ResearchNon-viral Gene Delivery

77. Manipulation of lipoplex concentration at the cell surface boosts transfection efficiency in hard-to-transfect cells

Sara Palchetti, Daniela Pozzi, Cristina Marchini, Augusto Amici, Cristina Andreani, Caterina Bartolacci, Luca Digiacomo, Valentina Gambini, Francesco Cardarelli, Carmine Di Rienzo, Giovanna Peruzzi, Heinz Amenitsch, Rocco Palermo, Isabella Scrapanti, Giulio Caracciolo

Nonviral DNA delivery remains inefficient, and the barriers limiting transfection are incompletely understood. The study investigates multicomponent cationic liposome/DNA lipoplexes to identify an additional transfection barrier and improve delivery, especially in hard-to-transfect cells. MC cationic liposomes: 78.8…

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Biomacromolecules2016ResearchNon-viral Gene Delivery

78. Highly Branched Poly(#-amino esters) for Non-Viral Gene Delivery High Transfection Efficiency and Low Toxicity Achieved by Increasing Molecular Weight

Yongsheng Gao, Jian-Yuan Huang, Jonathan O’keeffe Ahern, Lara Cutlar, Dezhong Zhou, Feng‐huei Lin, Wenxin Wang

Nonviral gene delivery vectors suffer from an “efficacy–toxicity paradox”: high transfection efficiency usually comes with high cytotoxicity, while low-toxicity vectors often transfect poorly. For cationic polymers such as PEI, PAMAM, and PDMAEMA, increasing molecular weight typically improves transfection but also…

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Acta Biomaterialia2016ResearchNon-viral Gene Delivery

79. Synthesis and application of poly(ethylene glycol)-co-poly(β-amino ester) copolymers for small cell lung cancer gene therapy

Kim, J., Kang, Y., Tzeng, S. Y., & Green, J. J. (2016).

Small cell lung cancer (SCLC) has high recurrence and mortality, and new therapies are needed. PBAE/DNA polyplexes are effective nonviral vectors but aggregate in physiological and acidic buffers, limiting therapeutic utility. PEGylation can improve stability but often reduces cellular uptake and transfection—the…

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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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2016ReviewDrug Delivery

81. Nanostructured Lipid Carriers A Novel Platform for Chemotherapeutics

Rizwanullah M, Ahmad J, Amin S

Conventional cancer chemotherapy suffers from poor drug specificity, narrow therapeutic windows, high dose-limiting toxicity, and multidrug resistance (MDR). Nanostructured lipid carriers (NLCs) are biocompatible, biodegradable lipid-based nanoparticles that can improve solubility, stability, targeting, and…

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Nature2016ReviewNon-viral Gene Delivery

82. Mimicking biological functionality with polymers for biomedical applications

Green, J. J.; Elisseeff, J. H

Biomaterials must increasingly do more than simply avoid rejection; they must actively mimic biological structures and functions to direct cell behaviour, modulate immunity, and repair tissues. This review addresses how biologically inspired polymers can be designed for local tissue engineering, systemic therapeutic…

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Advanced Drug Delivery Reviews2016ReviewNon-viral Gene Delivery

83. PEGylation as a strategy for improving nanoparticle-based drug and gene delivery

Suk, J. S.; Xu, Q.; Kim, N.; Hanes, J.; Ensign, L. M

PEGylation is widely used to improve nanoparticle drug and gene delivery by reducing aggregation, opsonisation, and phagocytic clearance, thereby prolonging circulation. However, the field lacks a consolidated understanding of how PEG molecular weight, surface density, conformation, core properties, administration…

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International Journal of Nanomedicine2015ResearchNon-viral Gene Delivery

84. An Efficient Method for In Vitro Gene Delivery via Regulation of Cellular Endocytosis Pathway

Jing Luo, Caixia Li, Jianlin Chen, Gang Wang, Rong Gao, Zhongwei Gu

Nonviral gene carriers (cationic polymers and lipids) are promising alternatives to viral vectors, but their transfection efficiencies are much lower. While extensive efforts have focused on chemical modification of gene delivery materials, simple optimisation of the transfection procedure itself—beyond standard…

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Proceedings of the National Academy of Sciences (PNAS)2015ResearchNon-viral Gene Delivery

85. Dynamin 2-dependent endocytosis sustains T-cell receptor signaling and drives metabolic reprogramming in T lymphocytes

Tim Willinger, Matthew Staron, Shawn M. Ferguson, Pietro De Camilli, Richard A. Flavell

Prolonged T-cell receptor (TCR) signalling is required for T lymphocyte proliferation, yet TCR ligation paradoxically causes internalisation and down-modulation of the TCR from the cell surface. It is unknown how TCR signalling is sustained for many hours despite lower surface expression. Understanding whether TCR…

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International Journal of Nanomedicine2015ResearchNon-viral Gene Delivery

86. Targeted lung cancer therapy: preparation and optimization of transferrin-decorated nanostructured lipid carriers as novel nanomedicine for co-delivery of anticancer drugs and DNA

Zhenyu Shao, Jingyu Shao, Bingxu Tan, Shanghai Guan, Zhulong Liu, Zengjun Zhao, Fangfang He, Jian Zhao

Lung cancer chemotherapy is limited by multidrug resistance, severe adverse effects, and poor tumour targeting. A nanocarrier that actively targets lung cancer cells and co-delivers a cytotoxic drug plus therapeutic DNA could improve efficacy and reduce off-target toxicity. Tf₅ₖ-PTX-DNA-NLC: ~133 nm, zeta +19 mV…

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ACS Nano2015ResearchNon-viral Gene Delivery

87. Polymeric Nanoparticles for Non-Viral Gene Therapy Extend Brain Tumor Survival In Vivo

Antonella Mangraviti, Stephany Yi Tzeng, Kristen Lynn Kozielski, Yuan Wang, Yike Jin, David Gullotti, Mariangela Pedone, Nitsa Buaron, Ann Liu, David R. Wilson, Sarah K. Hansen, Fausto J. Rodriguez, Guo-Dong Gao, Francesco Dimeco, Henry Brem, Alessandro Olivi, Betty Tyler, Jordan J. Green

Glioblastoma remains highly lethal with poor prognosis despite surgery, radiation, and chemotherapy. Gene therapy is a promising alternative, but viral vectors carry safety risks. Non-viral polymeric nanoparticles are safer but have historically shown poor in vivo efficacy. A biodegradable poly(beta-amino ester)…

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Journal of Controlled Release.2015ReviewNon-viral Gene Delivery

88. 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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Macromolecular Bioscience2015ReviewNon-viral Gene Delivery

89. Defined Polymeric Materials for Gene Delivery

He, D.; Wagner, E

Polymeric gene delivery carriers such as PEI and poly(L-lysine) are heterogeneous and polydisperse, making site-specific multifunctional modification, structure–activity relationship studies, and reproducible clinical manufacturing difficult. This review argues that precisely defined polymeric materials — dendrimers…

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Journal of Controlled Release2015ReviewNon-viral Gene Delivery

90. 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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2015ReviewNon-viral Gene Delivery

91. Polymeric Nanocarriers for Non-Viral Gene Delivery

Li, L.; Wei, Y.; Gong, C

Viral vectors are efficient but carry risks of immunogenicity, inflammatory reactions, recombination, limited DNA cargo capacity, and manufacturing difficulties. Non-viral polymeric vectors are safer and easier to produce, but their transfection efficiency remains much lower than viral vectors because of multiple…

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Cancer Biology & Therapy2014ResearchNon-viral Gene Delivery

92. Liposome-Coated Lipoplex-Based Carrier for Antisense Oligonucleotides

Paulina Wyrozumska, Justyna Meissner, Monika Toporkiewicz, Marta Szarawarska, Kazimierz Kuliczkowski, Maciej Ugorski, Marta A. Walasek & Aleksander F. Sikorski

Most cancer gene therapy research has focused on solid tumours, while blood cancers such as leukaemia and lymphoma have received less attention. There is a need for a lipid carrier that protects antisense oligonucleotides or plasmid DNA from enzymatic degradation, is stable in serum, has low toxicity, and can…

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Polymers for Advanced Technologies (Polym. Adv. Technol.)2014ResearchNon-viral Gene Delivery

93. Bioreducible Polyspermine as Less Toxic and Efficient Gene Carrier

Hongsuk Park, Joseph W. Nichols, Han Chang Kang, You Han Bae

Bioreducible polymers are attractive as gene carriers because they can reduce cytotoxicity compared with non-degradable cationic polymers such as polyethyleneimine (PEI). However, the safety and transfection efficiency of spermine-based bioreducible polyspermines had not been directly compared with linear PEI (lPEI)…

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Acta Biomaterialia2014ResearchNon-viral Gene Delivery

94. 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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Bioconjugate Chemistry2014ResearchNon-viral Gene Delivery

95. Differential Polymer Structure Tunes Mechanism of Cellular Uptake and Transfection Routes of Poly(β-amino ester) Polyplexes in Human Breast Cancer Cells

Kim, J., Sunshine, J. C., & Green, J. J. (2014).

Nonviral gene delivery must overcome cellular uptake, endosomal escape, and nuclear transport, but the cellular uptake mechanism of poly(β-amino ester) (PBAE) polyplexes was unknown. Triple-negative breast cancer (TNBC) also lacks effective targeted therapies, making improved nonviral gene delivery strategies…

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Nature Reviews Genetics2014ReviewNon-viral Gene Delivery

96. 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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