Accounts of Chemical Research2008ReviewNon-viral Gene Delivery
Green, J. J.; Langer, R.; Anderson, D. G
Gene therapy has enormous therapeutic potential, but delivery remains the central bottleneck. Viral vectors are efficient but carry serious safety, immunogenicity, manufacturing, and cargo-capacity limitations. Nonviral vectors are safer and more scalable but typically orders of magnitude less effective. This Account…
Read the article →2007ReviewNon-viral Gene Delivery
Li W, Szoka Fc Jr
Systemic non-viral gene delivery is limited by rapid clearance, toxicity, and poor target-cell access. Cationic lipid–DNA lipoplexes are effective in vitro but perform poorly in vivo because they aggregate with serum proteins, are cleared by the reticuloendothelial system (RES), and accumulate mainly in lung and…
Read the article →Expert Opinion on Therapeutic Patents2006ReviewNon-viral Gene Delivery
Mcneil Se, Perrie Y
Cationic liposomes have been developed for gene delivery for almost 20 years, but despite extensive effort, there has been limited progress toward an effective pharmaceutical product. Their transfection efficiency remains insufficient, and most clinical trials still rely on more potent viral vectors despite viral…
Read the article →Nature Materials2006ReviewNon-viral Gene Delivery
Putnam D
Synthetic polymer-based gene delivery vectors have been studied for two decades but remain far less efficient than viral vectors. The review addresses the need for improved design criteria and new synthetic strategies across nano-, micro-, and macroscale delivery systems to overcome formulation, organism-level, and…
Read the article →Journal of Controlled Release2006ReviewNon-viral Gene Delivery
Lv H, Zhang S, Wang B, Cui S, Yan J
Cationic lipids and polymers are promising non-viral gene delivery vectors because they are non-immunogenic, easy to produce, and not oncogenic, but toxicity remains a major obstacle to their clinical application. The review evaluates structural features of cationic compounds and summarises toxicity–structure…
Read the article →Gene Therapy2005ReviewNon-viral Gene Delivery
Dean Da, Strong Dd, Zimmer We.
Nonviral gene delivery is limited by multiple extracellular and intracellular barriers, but the nuclear envelope remains a major unresolved barrier, especially in nondividing cells. Plasmids must enter the nucleus to be transcribed, yet the mechanisms of plasmid nuclear import are much less understood than protein…
Read the article →Expert Opinion on Drug Delivery2005ReviewNon-viral Gene Delivery
Sérgio Simões, Ana Da Silva Filipe, Henrique Faneca, Miguel Mano, Nuno Penacho, Nejat Düzgüneş, Maria C. Pedroso De Lima
Cationic liposome–DNA complexes (lipoplexes) are a potentially viable non-viral alternative to viral vectors for therapeutic gene delivery, but they suffer from limited delivery/gene expression efficiency, toxicity at higher concentrations, adverse interactions with serum and cell surfaces, and poor tissue access…
Read the article →Nature Reviews Drug Discovery2005ReviewNon-viral Gene Delivery
Pack, D. W.; Hoffman, A. S.; Pun, S.; Stayton, P. S
The lack of safe and efficient gene-delivery methods remains a limiting obstacle to human gene therapy. Synthetic vectors are safer than recombinant viruses but generally lack sufficient efficacy, so polymers must be rationally designed to overcome extracellular and intracellular delivery barriers. PEI: Only 15–20%…
Read the article →Trends in Immunology2002ReviewNon-viral Gene Delivery
James Harris, Dirk Werling, Jayne C. Hope, Geraldine Taylor, Chris J. Howard
Caveolae and caveolin are cholesterol-rich membrane microdomains involved in endocytosis, cholesterol regulation, and signal transduction, but their presence and function in immune cells have been contentious. This review addresses the need to clarify the distribution and roles of caveolae/caveolin in mammalian…
Read the article →2001ReviewNon-viral Gene Delivery
Kircheis, R.; Wightman, L.; Wagner, E
PEI is one of the most effective non-viral gene delivery polymers, but its efficacy, toxicity, and in vivo behaviour depend strongly on molecular weight, branching, particle size, surface charge, and modifications. This review addresses how PEI/DNA complexes can be modified with targeting ligands, shielding agents…
Read the article →1999ReviewNon-viral Gene Delivery
Godbey, W. T.; Wu, K. K.; Mikos, A. G
Gene therapy still lacks safe and efficient delivery vectors. PEI has emerged as a promising non-viral polycation because it condenses DNA and mediates endosomal escape, but its transfection mechanism, targeting potential, and toxicity require clearer understanding. This review summarises PEI chemistry, PEI/DNA…
Read the article →1989ReviewNon-viral Gene Delivery
Felgner Pl, Ringold Gm
Existing polynucleotide delivery methods—calcium phosphate, DEAE-dextran, electroporation, retroviral vectors—had limitations in convenience, reproducibility, efficiency, and applicability across cell types. A simple, broadly effective cationic liposome reagent was needed for DNA and RNA transfection of mammalian…
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