Current Gene Therapy

Ignacio Anegon
Director INSERM UMR 1064-Center for Research in Transplantation and Immunology
CHU de Nantes. 30, boulevard
Nantes
France

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Production of Non Viral DNA Vectors

Author(s): Martin Schleef, Markus Blaesen, Marco Schmeer, Ruth Baier, Corinne Marie, George Dickson, Daniel Scherman.

Abstract:

After some decades of research, development and first clinical approaches to use DNA vectors in gene therapy, cell therapy and DNA vaccination, the requirements for the pharmaceutical manufacturing of gene vectors has improved significantly step by step. Even the expression level and specificity of non viral DNA vectors were significantly modified and followed the success of viral vectors. The strict separation of “viral” and “non-viral” gene transfer are historic borders for scientists and we will show that both fields together are able to allow the next step towards successful prevention and therapy. Here we summarize the features of producing and modifying these non-viral gene vectors to ensure the required quality to modify cells and to treat human and animals.

Keywords: Contract manufacturing, gene and cell therapy, minicircle, pFAR, Plasmid DNA, quality assurance and control, vaccination, vector production, aerosolization, hydrodynamic delivery, block co-polymers, cationic lipids, Polymeric Micelles, Enhanced Permeability and Retention (EPR) effect, Photochemical internalization (PCI), photosensitiser (PS), Lipofection, Gene Gun, Hydrodynamic Gene Delivery, Jet Injection, Particle Bombardment, cystic fibrosis, emphysema, polymer-DNA complexes, Electroporative Transfection (Electrotransfer), electrochemotherapy (ECT), Magnetofection, Sonoporation, Virus Mediated DNA Delivery, Ebola, Stem Cells, wild type, Bacillus subtilis, E. coli, Streptomyces coelicolor, ColE1, RNAII, Rom, ParA, ParB, MCS, pUC21, MCB, WCB, QC assays, bioinformatics, osmolarity, BSE, TSE, pepsin, pancreatin, trypsin, semi-generic, fed-batch techniques, pBR322, lipopolysaccarides, Ribonucleases (RNases), hydrolyse phosphodiester bonds, shear force, cleared lysate, CpG-free plasmid, zero-CpG-plasmid, Bioburden, Biosafe Miniplasmids, pCOR, R6K, thyA gene, dapD, lac operon, IPTG, lacOS, pORT, SV40-promoter

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Article Details

VOLUME: 10
ISSUE: 6
Year: 2010
Page: [487 - 507]
Pages: 21
DOI: 10.2174/156652310793797711
Price: $58