Journal Article10.1038/NRG3763
Non-viral vectors for gene-based therapy
Hao Yin,Rosemary Lynn Kanasty,Ahmed A. Eltoukhy,Arturo J. Vegas,J. Robert Dorkin,Daniel G. Anderson +5 more
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TL;DR: The biological barriers to gene delivery in vivo are introduced and recent advances in material sciences, nanotechnology and nucleic acid chemistry that have yielded promising non-viral delivery systems are discussed, some of which are currently undergoing testing in clinical trials.
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Abstract: Gene-based therapy is the intentional modulation of gene expression in specific cells to treat pathological conditions This modulation is accomplished by introducing exogenous nucleic acids such as DNA, mRNA, small interfering RNA (siRNA), microRNA (miRNA) or antisense oligonucleotides Given the large size and the negative charge of these macromolecules, their delivery is typically mediated by carriers or vectors In this Review, we introduce the biological barriers to gene delivery in vivo and discuss recent advances in material sciences, nanotechnology and nucleic acid chemistry that have yielded promising non-viral delivery systems, some of which are currently undergoing testing in clinical trials The diversity of these systems highlights the recent progress of gene-based therapy using non-viral approaches
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Citations
Peptide-grafted dextran vectors for efficient and high-loading gene delivery
Yang Hu,Huifeng Wang,Hai-Qing Song,Megan Young,Fan Yaqian,Fu-Jian Xu,Xinjian Qu,Xia Lei,Ying Liu,Gang Cheng +9 more
TL;DR: It is unveiled that histidine is critical for both DNA condensation and the accurate control of endosomal escape and the tunable D-RxHy platform not only demonstrates promising potential for therapeutic purposes but can also be used as a tool to elucidate the molecular mechanism of polymer-based transfection.
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Non-viral vectors based on magnetoplexes, lipoplexes and polyplexes for VEGF gene delivery into central nervous system cells.
Ilia Villate-Beitia,Gustavo Puras,Cristina Soto-Sánchez,Mireia Agirre,Edilberto Ojeda,Jon Zarate,Eduardo Fernández,José Luis Pedraz +7 more
TL;DR: Three different non-viral vectors based on magnetic, cationic lipid and polymeric nanoparticles complexed to the phVEGF165aIRESGFP plasmid, which codifies the VEGF protein -extracellular- and the green fluorescent protein (GFP) -intracellular-.
24
Genetically engineered mesenchymal stem cell therapy using self-assembling supramolecular hydrogels.
Byung Woo Hwang,Su Jin Kim,Kyeng Min Park,Hyemin Kim,Junseok Yeom,Jeong-A Yang,Hyeonseon Jeong,Hyuntae Jung,Kimoon Kim,Young Chul Sung,Sei Kwang Hahn +10 more
TL;DR: The genetic engineering of MSCs, recent progress of self-assembling supramolecular hydrogels, and their applications to cell therapy for intractable diseases and tissue regeneration are described.
24
Efficient delivery of DNA into bovine preimplantation embryos by multiwall carbon nanotubes.
Michele Munk,Luiz Orlando Ladeira,Bruno Campos de Carvalho,Luiz Sérgio de Almeida Camargo,Nádia Rezende Barbosa Raposo,R. V. Serapião,C. C. R. Quintão,Saulo R. Silva,Jaqueline S. Soares,Ado Jorio,Humberto M. Brandão +10 more
TL;DR: It is confirmed that multiwall carbon nanotubes (MWNTs) can cross the PZ and delivery of pDNA into in vitro-fertilized bovine embryos and the degeneration rate and the expression of genes associated to cell viability were not affected in embryos exposed to MWNTs.
Clinical Evaluations of Toxicity and Efficacy of Nanoparticle-Mediated Gene Therapy
TL;DR: This review highlights clinical trials that examined cationic nanocarrier-mediated gene therapy as well as discusses both the toxicity and efficacy of nanoccarrier-based therapeutics.
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