Towards safe, non-viral therapeutic gene expression in humans.
The potential dangers of using viruses to deliver and integrate DNA into host cells in gene therapy have been poignantly highlighted in recent clinical trials. Safer, non-viral gene delivery approaches have been largely ignored in the past because of their inefficient delivery and the resulting transient transgene expression. However, recent advances indicate that efficient, long-term gene expression can be achieved by non-viral means. In particular, integration of DNA can be targeted to specific genomic sites without deleterious consequences and it is possible to maintain transgenes as small episomal plasmids or artificial chromosomes. The application of these approaches to human gene therapy is gradually becoming a reality.
Glover
Dominic J
DJ
Nuclear Signalling Laboratory, Department of Biochemistry and Molecular Biology, Monash University, Victoria 3800, Australia.
Lipps
Hans J
HJ
Jans
David A
DA
D022201:Chromosomes, Artificial; D015870:Gene Expression; D018390:Gene Targeting; D018014:Gene Transfer Techniques; D005814:Genes, Viral; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006801:Humans; D008081:Liposomes; D010957:Plasmids; D011995:Recombination, Genetic; D019076:Transgenes
D008081:Liposomes