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PMID: 11020345 Published · ppublish English Journal Article Research Support, Non-U.S. Gov't Review

Development of biomaterials for gene therapy.

Han S, Mahato RI, Sung YK, Kim SW

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.

MeSH Terms
Biocompatible Materials Biotransformation Cation Exchange Resins Drug Carriers/administration & dosage,pharmacology Drug Delivery Systems/methods Drug Stability Genetic Therapy/methods Humans Plasmids/genetics,pharmacokinetics Polymers/therapeutic use Transfection/methods
Chemicals
Biocompatible Materials Cation Exchange Resins Drug Carriers Polymers
Authors & Affiliations
4 authors, click to expand affiliations / ORCID
Han S
Center for Controlled Chemical Delivery (CCCD), University of Utah, Salt Lake City, Utah, 84112, Korea.
Mahato R I
Sung Y K
Kim S W
Article Info
Journal
Molecular therapy : the journal of the American Society of Gene Therapy
Abbr.
Mol Ther
ISSN
1525-0016
Published
2000-10-00
Pages
302-17
Language
English
Region
United States
NLM ID
100890581
Subset
IM
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