Acta Pharmacologica Sinica2010ResearchNon-viral Gene Delivery
Jian Li, Ying-Zi He, Wen Li, Yun-Zhen Shen, Yu-Ru Li, Yun-Feng Wang
Non-viral gene delivery systems offer advantages in safety and ease of preparation over viral vectors, but suffer from limited transfection efficiency. While polymer-lipid hybrid nanoparticles (PLN) have shown promise as non-viral vectors, the incorporation of PEGylated lipids to enhance circulation time and…
Read the article →2010ResearchNon-viral Gene Delivery
Kai K. Ewert, Alexandra Zidovska, Ayesha Ahmad, Nathan F. Bouxsein, Heather M. Evans, Christopher S. Mcallister, Charles E. Samuel, Cyrus R. Safinya
Cationic lipid–nucleic acid (CL-NA) complexes are promising non-viral vectors for gene delivery and siRNA-mediated silencing, but their transfection and silencing efficiencies remain low compared with viral vectors. A major gap is poor mechanistic understanding of how complex structure, membrane charge density, and…
Read the article →Proceedings of the National Academy of Sciences of the United States of America2010ResearchNon-viral Gene Delivery
Kevin T. Love, Kerry P. Mahon, Christopher G. Levins, Kathryn A. Whitehead, William Querbes, J. Robert Dorkin, June Qin, William Cantley, Liu Liang Qin, Timothy Racie, Maria Frank-Kamenetsky, Ka Ning Yip, Rene Alvarez, Dinah W. Y. Sah, Antonin De Fougerolles, Kevin Fitzgerald, Victor Koteliansky, Ak
Existing siRNA delivery systems required high doses (1 mg/kg) for hepatic gene silencing. A more potent, well-tolerated lipid-like delivery material was needed to enable low-dose RNAi therapeutics and multi-gene silencing in vivo. C12-200 enabled Factor VII silencing at doses below 0.01 mg/kg in mice, 100-fold more…
Read the article →Journal of Internal Medicine2010ReviewNon-viral Gene Delivery
Schroeder A, Levins Cg, Cortez C, Langer R, Anderson Dg
RNAi has strong therapeutic potential, but clinical translation of siRNA is limited by delivery. siRNA is large, negatively charged, susceptible to nuclease degradation, poorly taken up by cells, and must reach the cytoplasm to engage RISC. Safe and effective lipid-based delivery systems are needed to protect siRNA…
Read the article →Journal of drug targeting2008ReviewDrug Delivery
Parveen, S.; Sahoo, S. K
Conventional cancer chemotherapy suffers from poor tumour selectivity, high required doses, systemic toxicity, rapid drug elimination, and multidrug resistance. Polymeric nanoparticles (NPs) are reviewed as a strategy to improve targeted delivery, controlled release, and therapeutic efficacy while reducing adverse…
Read the article →Journal of Controlled Release2007ResearchNon-viral Gene Delivery
Ayumi Sato, Sung Won Choi, Miwa Hirai, Asako Yamayoshi, Rui Moriyama, Takeshi Yamano, Motoki Takagi, Arihiro Kano, Akira Shimamoto, Atsushi Maruyama
Systemic siRNA therapeutics are limited by poor cell membrane permeability, rapid nuclease degradation, and renal clearance. A carrier with long circulatory half-life is needed for effective RNAi and potential tumour targeting via the enhanced permeation and retention (EPR) effect. Existing hydrophilic…
Read the article →Bioorganic & Medicinal Chemistry (inferred from journal style; exact journal not stated in supplied excerpt)2007ResearchNon-viral Gene Delivery
Masato Yamagata, Takahito Kawano, Kouhei Shiba, Takeshi Mori, Yoshiki Katayama, Takuro Niidome
Gene carriers are often studied individually under different assay conditions, making direct structure–function comparisons difficult. The study compared dendritic poly(L-lysine) (KG6) and linear poly(L-lysine) (PLL) within the same experimental system to identify how carrier architecture affects DNA complex…
Read the article →Journal of Controlled Release2006ResearchNon-viral Gene Delivery
Katas H, Alpar Ho
siRNA has therapeutic potential but is limited by poor cellular uptake, rapid nuclease degradation, and poor blood stability. Chitosan was explored as a non-viral siRNA vector because of its low toxicity, biodegradability, biocompatibility, and cationic nature, but its use for siRNA delivery had not been…
Read the article →Biomaterials2005ResearchDrug Delivery
Norbert Dinauer, Sabine Balthasar, Carolin Weber, Jörg Kreuter, Klaus Langer, Hagen Von Briesen
Antibody-based therapeutics have shown promise in targeting specific cell types, but their efficacy can be enhanced by conjugation to drug carriers. Nanoparticles as drug carriers offer advantages including high drug loading capacity, controlled release, and protection from degradation. However, achieving selective…
Read the article →Synthesis of Poly(β-amino esters) Optimized for Highly Effective Gene Delivery.2003ResearchNon-viral Gene Delivery
Akinc, A., Anderson, D. G., Lynn, D. M., & Langer, R. (2003).
Nonviral polymeric gene delivery vectors need systematic optimisation of nonstructural factors—polymer molecular weight, chain end-group, and polymer/DNA ratio—that strongly influence transfection, cytotoxicity, and DNA complexation. These factors had not been comprehensively studied together for poly(β-amino…
Read the article →Journal of Biological Chemistry2001ResearchNon-viral Gene Delivery
Ge Liu, Maria Molas, Gregory A. Grossmann, Murali Pasumarthy, Jose C. Perales, Mark J. Cooper, Richard W. Hanson
Receptor-mediated gene transfer using ligand–poly-L-lysine–DNA complexes requires DNA to be condensed into small particles for efficient cellular uptake and expression. However, the relationship between NaCl concentration, the mode of poly-L-lysine binding to DNA, the resulting condensed DNA structure, and…
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…
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Peng Zhang, Qinan Xu, Xinfang Li, Youxiang Wang
Non-viral gene vectors must overcome multiple extracellular and intracellular barriers: stability in blood, efficient cellular uptake, endosomal escape, and intracellular gene release. Stimuli-responsive carriers can address some barriers, but a carrier capable of overcoming multiple barriers simultaneously is still…
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Qiu T, Gu P, Wusiman A, Ni H, Xu S, Zhang Y, Zhu T, He J, Liu Z, Hu Y, Liu J, Wang D
Conventional adjuvants such as aluminium and Freund’s adjuvants have limitations, including poor cell-mediated immunity and local toxicity. Ginseng stem-leaf saponins (GSLS) have immunomodulatory/adjuvant activity, but improved delivery systems are needed. Cubosomes are stable lipid cubic liquid crystalline…
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