Molecular design of functional polymers for gene therapy
- 作者：Ji Hoon Jeong ; Sung Wan Kim ; Tae Gwan Park
- 关键词：Gene delivery ; Cationic polymers ; Nonviral carriers ; Polylexes
- 刊名：Progress in Polymer Science
- 出版年：2007
- 期刊代码：90_00796700
- 类别：ms
- 出版时间：November 2007
- 卷：32
- 期：11
- 页码：1239-1274
- 文件大小：1165 K
摘要
In the post genomic era, massive genetic information of the human being accelerates the identification of target genes for treating various diseases. A wide range of nucleic acid therapeutics have recently emerged as powerful new drug entities for treatment of gene-related diseases and for lead validation in the drug discovery process. The absence of an efficient delivery system for genetic drugs has been identified as a major hurdle for clinical applications. Various bio-mimicking polymers have been considered as potential nonviral carriers for gene therapy due to their safety in clinical use, despite their lower gene-transduction capacity compared to virus-based gene carriers that hold risk of potential immunogenicity and endogenous virus recombination. Improving specificity toward target cells and intracellular gene-transferring efficiency will be a prerequisite for successful clinical application of polymeric gene carriers. Multifunctional polymeric gene delivery carriers should be rationally designed at the molecular level for effective human gene therapy.