Nature Communications2017ResearchNon-viral Gene Delivery
H.F. Moffett, M.E. Coon, S. Radtke, S.B. Stephan, L. Mcknight, A. Lambert, B.L. Stoddard, H.P. Kiem, M.T. Stephan
Therapies based on genetically engineered immune cells (CAR T cells, HSCs) are transforming medicine, but the viral and electroporation methods used to create these cytoreagents are complex, expensive, and time-consuming. There is a growing need for transient "hit-and-run" genetic programming (transcription factors…
Read the article →Nature Nanotechnology2017ResearchNon-viral Gene Delivery
Tyrel T. Smith, Sirkka B. Stephan, Howell F. Moffett, Laura E. Mcknight, Weihang Ji, Diana Reiman, Emmy Bonagofski, Martin E. Wohlfahrt, Smitha P. S. Pillai, Matthias T. Stephan
Adoptive T cell therapy with CAR-engineered T cells has shown impressive clinical results, but the ex vivo manufacturing process is complex, expensive, and requires specialised facilities, limiting widespread application. A method to programme circulating T cells directly in vivo with tumour-recognising capabilities…
Read the article →2017ResearchNon-viral Gene Delivery
Petra Furedi, Zsofia Edit Papay, Kristof Kovacs, Borbala Dalmadi Kiss, Krisztina Ludanyi, Istvan Antal, Imre Klebovich
Topical fungal infections, including ocular mycoses, are increasing, and voriconazole (VCZ) is an effective antifungal but has poor water solubility, limiting liquid ophthalmic formulations. Lipid-based nanoparticles (LNPs/SLNs) may improve VCZ solubility, loading, and topical delivery. Optimised formulation (SLN2)…
Read the article →Biomaterials2017ResearchNon-viral Gene Delivery
Yoshizaki Y, Yuba E, Sakaguchi N, Koiwai K, Harada A, Kono K
Efficient cancer vaccine carriers need both antigen delivery to dendritic cells (DCs) and DC activation. Previous pH-sensitive polymer-modified liposomes delivered antigen to DC cytosol but had limited adjuvant potency, and combining cationic lipid TRX with MPLA reduced antitumor effects, suggesting a need for…
Read the article →International Journal of Pharmaceutics2017ResearchNon-viral Gene Delivery
Genada Sinani, Melike Sessevmez, M. Koray Gök, Saadet Özgümüş, Alper Okyar, H. Oya Alpar, Erdal Cevher
Mucosal vaccination can stimulate both mucosal and systemic immunity, but free protein antigens generally induce poor systemic immune responses and require adjuvants. There is a need for a safe, effective nasal vaccine carrier that improves antigen stability, uptake, and immunogenicity, especially for poorly…
Read the article →Biomaterials2017ResearchNon-viral Gene Delivery
Shi Gn, Zhang Cn, Xu R, Niu Jf, Song Hj, Zhang Xy, Wang Ww, Wang Ym, Li C, Wei Xq, Kong Dl
Whole tumour cell lysates (TCL) are a broad source of tumour antigens for cancer vaccines, but soluble TCL is unstable, poorly taken up by dendritic cells (DCs), and inefficient at inducing cytotoxic T lymphocyte (CTL) responses. A delivery system was needed to protect TCL antigens, target DCs, and enhance antigen…
Read the article →Journal of Controlled Release.2017ReviewNon-viral Gene Delivery
Tapeinos C, Battaglini M, Ciofani G
Delivery of therapeutics to the CNS is severely limited by the blood–brain barrier (BBB). Polymeric and inorganic nanoparticles have shown utility but face toxicity, scalability, and organic-solvent concerns. Solid lipid nanoparticles (SLNs) and nanostructured lipid carriers (NLCs) are biocompatible lipid-based…
Read the article →2017ReviewDrug Delivery
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…
Read the article →Materials2017ReviewNon-viral Gene Delivery
Sun, X.; Wu, G.; Xu, C.; Ye, Q.; Wang, C
Periodontitis causes irreversible destruction of periodontal tissues, and regeneration requires biomaterials with suitable mechanical, degradation, and biological properties. PLGA is attractive for periodontal regeneration because of its biocompatibility, tunable degradation, and processability, but its…
Read the article →Drug Development and Industrial Pharmacy2016ResearchDrug Delivery
Zhihe Lu, Jingrong Su, Zhengrong Li, Yuzhu Zhan, Decai Ye
Gemcitabine (GEM) monotherapy for pancreatic cancer has poor tumour specificity, short circulation half-life, and drug resistance. Baicalein (BCL) can downregulate Bcl-2/Mcl-1 and sensitise tumour cells, but it has poor solubility, low bioavailability, and rapid metabolism. A targeted co-delivery system was needed to…
Read the article →Nanomedicine: Nanotechnology, Biology, and Medicine2016ResearchNon-viral Gene Delivery
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…
Read the article →Molecular Therapy2016ResearchNon-viral Gene Delivery
Ans De Beuckelaer, Charlotte Pollard, Sandra Van Lint, Kenny Roose, Lien Van Hoecke, Thomas Naessens, Vimal Kumar Udhayakumar, Muriel Smet, Niek Sanders, Stefan Lienenklaus, Xavier Saelens, Siegfried Weiss, Guido Vanham, Johan Grooten & Stefaan De Koker
mRNA vaccines are promising for inducing cytolytic CD8⁺ T cell responses, and lipoplex carriers improve mRNA delivery to dendritic cells. However, the innate factors that regulate T cell induction by mRNA lipoplex vaccines remain unresolved. In particular, type I interferons (IFNs) are induced by mRNA but can either…
Read the article →Drug Delivery2016ResearchDrug Delivery
Jie Qu, Liangqiao Zhang, Zhihua Chen, Guohua Mao, Ziyun Gao, Xianliang Lai, Xingen Zhu, Jianming Zhu
Glioblastoma multiforme (GBM) has a poor prognosis due to limited drug delivery across the blood–brain barrier (BBB), therapeutic resistance, and recurrence. Nanocarriers may improve temozolomide (TMZ) delivery, but it is unclear which nanocarrier type—polymeric nanoparticles (PNPs), solid lipid nanoparticles (SLNs)…
Read the article →Biomacromolecules2016ResearchNon-viral Gene Delivery
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…
Read the article →Advanced Drug Delivery Reviews2016ReviewDrug Delivery
Niu Z, Conejos-Sánchez I, Griffin Bt, O’driscoll Cm, Alonso Mj
Peptide and protein therapeutics are highly potent but mostly require parenteral administration because they are degraded in the gastrointestinal tract, poorly permeate the intestinal epithelium, and have low oral bioavailability. Lipid-based nanocarriers are a promising strategy to enable oral peptide delivery by…
Read the article →Advanced Drug Delivery Reviews2015ReviewNon-viral Gene Delivery
Yunching Chen, Dong-Yu Gao, Leaf Huang
miRNAs can simultaneously regulate multiple cancer-related genes, making them attractive therapeutic agents or targets. However, efficient, specific, and safe systemic delivery of therapeutic miRNAs in vivo remains a major challenge due to rapid degradation, poor tumour penetration, immunotoxicity, endosomal…
Read the article →Proceedings of the National Academy of Sciences (PNAS)2015ResearchNon-viral Gene Delivery
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…
Read the article →Journal of Controlled Release 200 (2015ResearchNon-viral Gene Delivery
H. Akita Et Al.
Cationic gene carriers can mediate cellular uptake but often show poor post-nuclear transcription/translation, toxicity, and aggregation in blood. The study aimed to develop a systemically applicable, tumour-targeting gene carrier that is neutral in blood/cytosol but becomes cationic in endosomes for endosomal…
Read the article →International Journal of Nanomedicine2015ResearchDrug Delivery
Chun-Ying Qu, Min Zhou, Ying-Wei Chen, Mei-Mei Chen, Feng Shen, Lei-Ming Xu
5-Fluorouracil (5-FU) plus cisplatin (CDDP) is a first-line gastric cancer chemotherapy regimen, but it causes substantial toxicity and 5-FU has poor biopharmaceutical properties. A targeted nanoparticle platform could codeliver both drugs, improve synergy, and reduce systemic toxicity. Optimal HA coating…
Read the article →International Journal of Nanomedicine2015ResearchNon-viral Gene Delivery
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…
Read the article →2015ReviewNon-viral Gene 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…
Read the article →2015ReviewNon-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…
Read the article →Cold Spring Harbor Perspectives in Biology2014ReviewNon-viral Gene Delivery
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…
Read the article →2014ResearchDrug Delivery
Lopes Rm, Gaspar Mm, Pereira J, Eleuterio Cv, Carvalho M, Almeida Aj, Cruz Mem
Current leishmaniasis chemotherapy lacks options that combine high activity, low toxicity, and affordable cost. Oryzalin (ORZ), a dinitroaniline with in vitro antileishmanial activity, is limited by poor water solubility and low accumulation in diseased organs. Lipid-based nanocarriers may overcome these limitations…
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