What role can chemistry play in cationic liposome-based gene therapy research today?

Kostas Kostarelos, A.D Miller

Research output: Contribution to journalArticlepeer-review

Abstract

Gene therapy research is still in trouble owing to a paucity of acceptable vector systems to deliver nucleic acids to patients for therapy. Viral vectors are efficient but may be too dangerous for routine clinical use. Synthetic non-viral vectors are inherently much safer but are currently not efficient enough to be clinically viable. The solution for gene therapy lies with improved synthetic non-viral vectors based upon well-found platform technologies and a thorough understanding of the barriers to efficient gene delivery and expression (transfection) relevant to clinical applications of interest. Here we introduce and interpret synthetic non-viral vector systems through the ABCD nanoparticle structural paradigm that represents, in our view, an appropriate lens through which to view all synthetic, non-viral vector systems applicable to in vitro use or in vivo applications and gene therapy.
Original languageEnglish
Pages (from-to)71-118
Number of pages47
JournalAdvances in Genetics
Volume53
Issue number4
Publication statusPublished - 2005

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