Pharmaceutical perspectives of nonviral gene therapy.
The use of nonviral plasmid-based gene medicines represents an attractive in vivo gene transfer strategy that is simple and lacks many risks that are inherent to viral systems. Commercialization of gene medicines requires a thorough analysis of business opportunities, unmet clinical needs, competitive products under development, and issues related to intellectual property. Synthetic gene delivery systems are designed to control the location of a gene within the body by affecting distribution and access of a gene expression system to the target cell, and/or recognition by a cell surface receptor and uptake followed by intracellular and nuclear translocation. Plasmid-based gene expression systems are designed to control the level, fidelity, and duration of in vivo production of a therapeutic gene product. This review will provide insights into the potentials of plasmid-based gene therapy and critical evaluation of gene delivery sciences and clinical applications of gene medicines.
Mahato
R I
RI
Copernicus Therapeutics, Inc., Cleveland, Ohio 44106, USA. mahato@cgsys.com
Smith
L C
LC
Rolland
A
A
D000818:Animals; D001709:Biotechnology; D002986:Clinical Trials as Topic; D004274:DNA, Recombinant / Q000008:administration & dosage / Q000737:chemistry / Q000235:genetics; D016503:Drug Delivery Systems; D015870:Gene Expression; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000331:legislation & jurisprudence / Q000379:methods*; D005822:Genetic Vectors; D006801:Humans; D009369:Neoplasms / Q000628:therapy; D010957:Plasmids / Q000235:genetics; D014481:United States; D019444:Vaccines, DNA / Q000235:genetics
D004274:DNA, Recombinant; D019444:Vaccines, DNA