Human Gene Therapy* (as indicated by article context and references)2018ReviewNon-viral Gene Delivery
Yao Xiao, Kun Shi, Ying Qu, Bingyang Chu, Zhiyong Qian
In the past 10 years, many clinical nano-gene therapy trials have been discontinued due to poor efficacy and serious side effects. There is a critical need to design suitable gene delivery systems that can protect nucleic acids, enhance transfection efficiency, penetrate deep into tumours, and remain stable and safe…
Read the article →Nature Communications2018ResearchNon-viral Gene Delivery
Ewoud B. Compeer, Felix Kraus, Manuela Ecker, Gregory Redpath, Mayan Amiezer, Nils Rother, Philip R. Nicovich, Natasha Kapoor-Kaushik, Qiji Deng, Guerric P.B. Samson, Zhengmin Yang, Jieqiong Lou, Michael Carnell, Haig Vartoukan, Katharina Gaus, Jérémie Rossy
Polarized endocytic recycling of the T cell receptor (TCR) to the immunological synapse is essential for T cell activation, yet the cellular mechanisms that coordinate internalisation of surface receptors with sustained delivery back to the plasma membrane remain incompletely understood. The role of flotillins in…
Read the article →International Journal of Molecular Sciences2018ResearchNon-viral Gene Delivery
Simon A. B. Riedl, Alexander Raup, Patrick Kaiser, Christopher V. Synatschke, Valerie Jérôme, Ruth Freitag
Genetic modification of human T lymphocytes is critical for both basic research and emerging therapies (e.g., CAR-T cells), but established non-viral methods are highly inefficient. Linear polyethylenimine (l-PEI), the "gold standard" for non-viral transfection of mammalian cells, achieves only single-digit…
Read the article →Molecular Therapy2018ResearchNon-viral Gene Delivery
Daniel L. Jasinski, Hui Li, Peixuan Guo
Drugs with ideal pharmacokinetic profiles require long half-lives but little organ accumulation. Generally, these factors are contradictory: smaller particles lead to faster excretion and shorter half-lives, while larger particles lead to longer circulation but stronger organ accumulation and potential toxicity. The…
Read the article →Toxicology Research (RSC)2018ResearchNon-viral Gene Delivery
Shaohui Cui, Yueying Wang, Yan Gong, Xiao Lin, Yinan Zhao, Defu Zhi, Quan Zhou, Shubiao Zhang
Cationic lipids are widely used non-viral gene delivery vectors, but their toxicity remains a major bottleneck for clinical translation. Cytotoxicity is strongly associated with the cationic headgroup structure, yet the relationship between headgroup chemistry and toxic mechanisms has been rarely studied. IC50…
Read the article →Pharmaceutical Research2018ResearchNon-viral Gene Delivery
Francesca Sacchetti, Gaetano Marverti, Domenico D’arca, Leda Severi, Eleonora Maretti, Valentina Iannuccelli, Salvatore Pacifico, Glauco Ponterini, Maria Paola Costi, Eliana Leo
PEGylated pH-sensitive liposomes may improve delivery of anti-cancer peptides, but PEGylation can reduce cellular uptake and endosomal escape (“PEG dilemma”), and it was unknown whether PEGylated liposome components interfere with the intracellular mechanism of action of the delivered drug. Physicochemical…
Read the article →Molecular Pharmaceutics (Just Accepted Manuscript)2018ResearchNon-viral Gene Delivery
David P. Walsh, Robert D. Murphy, Angela Panarella, Rosanne M. Raftery, Brenton Cavanagh, Jeremy C. Simpson, Fergal J. O’brien, Andreas Heise, Sally-Ann Cryan
Tissue engineering needs efficient, biocompatible non-viral gene delivery to mesenchymal stem cells (MSCs), which are inherently refractory to transfection. Existing vectors have limitations: PEI is cytotoxic and linear poly(L-lysine) (L-PLL) has poor transfection efficiency. Transfection efficiency (pGFP, day 7)…
Read the article →Journal of Controlled Release2018ResearchNon-viral Gene Delivery
Brynn R. Olden, Yilong Cheng, Jonathan L. Yu, Suzie H. Pun
CAR T cell immunotherapy requires efficient ex vivo gene delivery to primary human T cells, but viral vectors have limitations including cost, cargo size, insertional mutagenesis risk, and manufacturing complexity. Non-viral cationic polymers are attractive alternatives but historically show low gene transfer…
Read the article →ACS Applied Bio Materials (Just Accepted Manuscript)2018ResearchNon-viral Gene Delivery
Chao Wang, Pengfei Sun, Gaina Wang, Pengcheng Yuan, Rongcui Jiang, Wenjun Wang, Wei Huang, Quli Fan
Dendritic cell (DC)-based vaccines require efficient antigen delivery, but free ovalbumin (OVA) is poorly taken up by DCs and is often degraded, sequestered, or exocytosed. Existing OVA delivery systems often suffer from low loading capacity (<10%), lack of imaging ability, or chemical modification that can impair…
Read the article →International Journal of Pharmaceutics2018ResearchNon-viral Gene Delivery
Haimanti Mandal, Sameer S. Katiyar, Rajan Swami, Varun Kushwah, Parmeshwar B. Katare, Anand Kumar Meka, Sanjay K. Banerjee, Amirali Popat, Sanyog Jain
Non-viral gene delivery vectors are safer than viral vectors but often suffer from lower transfection efficiency and cytotoxicity. ε-Poly-L-lysine (ε-PLL) is a naturally occurring, FDA GRAS cationic polymer with low molecular weight and potential for low toxicity, but its use as a pDNA nanocarrier for efficient in…
Read the article →Advanced Healthcare Materials (Adv. Healthcare Mater.)2018ResearchNon-viral Gene Delivery
Sungjin Min, Yoonhee Jin, Chen Yuan Hou, Jayoung Kim, Jordan J. Green, Taek Jin Kang, Seung-Woo Cho
Current cancer treatments (chemotherapy, radiotherapy) suffer from a lack of selectivity for tumour cells, causing severe side effects and therapeutic resistance. While gene therapy is a promising alternative, viral vectors pose safety risks (immunogenicity, tumorigenicity), and nonviral vectors often suffer from low…
Read the article →Nature Reviews Chemistry2018ReviewNon-viral Gene Delivery
Irene Lostalé-Seijo And Javier Montenegro
Gene therapy has enormous therapeutic potential, but clinical translation is limited by the delivery problem: nucleic acids are negatively charged, unstable, and must overcome multiple biological barriers to reach their intracellular site of action. Viral vectors are efficient but suffer from immunogenicity, limited…
Read the article →Bioscience Reports2017ReviewNon-viral Gene Delivery
Haiqing Bai, Gillian M. Schiralli Lester, Laura C. Petishnok, David A. Dean
Productive gene transfer requires plasmid DNA to not only enter cells but also traffic through the cytoplasm and cross the nuclear envelope. Most transfection strategies focus on membrane entry and promoter choice, but cytoplasmic trafficking and nuclear import are critical, rate-limiting barriers—especially because…
Read the article →PLOS ONE2017ResearchNon-viral Gene Delivery
Susanne Przybylski, Michaela Gasch, Anne Marschner, Marcus Ebert, Alexander Ewe, Gisa Helmig, Nadja Hilger, Stephan Fricke, Susanne Rudzok, Achim Aigner, Jana Burkhardt
Nanoparticle-based gene delivery methods are promising for transfecting immune cells, but their impact on basic immune functions such as proliferation—a cornerstone of cellular immunity—has not been systematically investigated. Understanding whether transfection reagents non-specifically influence immune cell…
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 →Colloids and Surfaces B: Biointerfaces2017ResearchNon-viral Gene Delivery
Pinnapireddy Sr, Duse L, Strehlow B, Schäfer J, Bakowsky U
Polyethylenimine (PEI)-based gene delivery is limited by cytotoxicity, rapid degradation, and low cellular uptake. A safer, more stable non-viral system was needed for both gene delivery and gene knockdown. Physicochemical properties: DDC liposomes ~198.7 nm, ζ = −30.9 mV; IPEI polyplexes ~166.2 nm, ζ = +19.1 mV…
Read the article →Journal of Biomedical Materials Research Part A2017ResearchNon-viral Gene Delivery
Wilson, D. R., Et Al. (2017).
Clinical translation of biomaterial-based gene delivery nanoparticles is limited by efficacy, safety, batch-to-batch consistency, scalable manufacturing, and long-term storage stability. Continuous microfluidic fabrication may overcome manufacturing and scale-up challenges for biodegradable PBAE/DNA polyplexes. DNA…
Read the article →2017ReviewNon-viral Gene Delivery
Jiménez Blanco Jl, Benito Jm, Ortiz Mellet C, García Fernández Jm.
Nonviral gene delivery lacks molecularly well-defined carriers suitable for rigorous structure–activity relationship (SAR) studies. Viral vectors raise immunogenicity, cost, and cargo-size concerns, while polymers, dendrimers, and cationic lipids are often polydisperse or supramolecularly ill-defined. The review…
Read the article →Gene Therapy2017ReviewNon-viral Gene Delivery
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…
Read the article →Advanced Drug Delivery Reviews2016ReviewNon-viral Gene Delivery
Jayoung Kim, Adam C. Mirando, Aleksander S. Popel, Jordan J. Green
Angiogenesis is normally balanced by pro- and anti-angiogenic factors, but imbalance leads to aberrant angiogenesis in ischaemia, tissue regeneration, cancer, and neovascular age-related macular degeneration (wet AMD). Gene therapy is a promising strategy to introduce exogenous nucleic acids that express or silence…
Read the article →Materials Science and Engineering C2016ResearchNon-viral Gene Delivery
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…
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 →