alexa Towards safe, non-viral therapeutic gene expression in humans.


Drug Designing: Open Access

Author(s): Glover DJ, Lipps HJ, Jans DA, Glover DJ, Lipps HJ, Jans DA

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Abstract 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. This article was published in Nat Rev Genet and referenced in Drug Designing: Open Access

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