A non-viral gene delivery system designed for clinical use.
Gene delivery can be accomplished using non-viral systems, and these have received increased attention These include ex vivo transfection of cells using an electric field to induce transient cell-membrane permeability (electroporation). This approach has the distinct advantage of not requiring the inclusion of a secondary agent (e.g. a lipid, viral package or carrier protein) any of which can be immunogenic or toxic. Available electroporation systems utilize a low volume (<1 mL) processing chamber and are open systems. The MaxCyte system employs a continuous flow design and can very rapidly process volumes ranging from 0.02 mL to >1 L. Transgenes for markers (eGFP) and functional proteins (e.g., cytokines, angiogenic factors) have been loaded in plasmids up to 14 kB in size. With appropriate application of pre- and post-processing cell manipulations, very satisfactory loading efficiencies and cell viability have been obtained. Cells can be processed with multiple plasmids, resulting in expression of the corresponding number of gene products. This capability has been considered for therapeutic and bioprocessing applications. The MaxCyte system was designed specifically for ex vivo clinical applications. The electrodes are manufactured of special materials and under precise conditions, in order to eliminate potential risks from electrolytic effects. The processing chamber and associated containers can be provided as disposable, sterile, closed (or functionally closed) systems-quite similar to the disposable harnesses used with cell separators. This system is thus suitable for integration into a current good manufacturing practice environment.
Fratantoni
J C
JC
MaxCyte, Rockville, MD 20850, USA.
Dzekunov
S
S
Singh
V
V
Liu
L N
LN
D002986:Clinical Trials as Topic; D018274:Electroporation / Q000295:instrumentation / Q000379:methods*; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D010957:Plasmids / Q000235:genetics*; D014162:Transfection / Q000379:methods*