Development of biomaterials for gene therapy

Sang Oh Han, Ram I. Mahato, Yong Kiel Sung, Sung Wan Kim

Research output: Contribution to journalReview article

332 Scopus citations

Abstract

Novel biocompatible polymeric gene carriers have been examined for their potential in treating various genetic and acquired diseases. The use of polymeric gene carriers may overcome the current problems associated with viral vectors in safety, immunogenicity, and mutagenesis. However, effective polymer-based gene therapy requires the control of cellular access and uptake, intracellular trafficking, and nuclear retention of plasmid DNA. Inefficient endosomal release, cytoplasmic transport, and nuclear entry of plasmids are currently limiting factors in the use of polymers for effective plasmid-based gene therapy. Therefore, several different polymeric gene carriers have been designed recently in an attempt to overcome these problems. This review explores the conceptual and experimental aspects of polymer-based gene delivery and presents an overview on the recent use of polymers to enhance the effectiveness of plasmid-based systems. Despite their current limitations, polymeric carriers have significant potential as commercially viable gene medicines.

Original languageEnglish (US)
Pages (from-to)302-317
Number of pages16
JournalMolecular Therapy
Volume2
Issue number4
DOIs
StatePublished - Oct 2000

Keywords

  • Biodegradable polymer
  • Biomaterials
  • Cytotoxicity
  • Functional group
  • Gene expression
  • Polymers

ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology
  • Genetics
  • Pharmacology
  • Drug Discovery

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