Biomedical Engineering Reference
In-Depth Information
CHAPTER 4
Functional Polymers for Gene
Delivery
d n 4 y 3 n g | 3
XUAN ZENG, REN-XI ZHUO AND
XIAN-ZHENG ZHANG*
Key Laboratory of Biomedical Polymers of Ministry of Education &
Department of Chemistry, Wuhan University, Wuhan 430072, P. R. China
* E-mail: xz-zhang@whu.edu.cn
4.1 Introduction
Gene therapy is an innovational approach for devastating inherited or
acquired disease treatment through delivering therapeutic genes to targeted
cells and replacing the disorder genes, where conventional therapy, such as
radiotherapy or chemotherapy, has met strong resistance. This therapeutic
process can be administrated in vivo or ex vivo (Figure 4.1). However, the
negatively charged cell membrane inhibits the entry of naked DNA due to its
electronegativity, and the unprotected DNA will be rapidly degraded by
nucleases present in plasma, so that most gene transfer is carried out using a
powerful gene delivery vehicle. Over the past two decades, gene therapy has
made great progress, especially in the development of effective gene vectors. 1
Some of these vectors have been successfully used in animal models and are
currently being tested in clinical trials to treat maladies such as cardiovascular
disease, cystic fibrosis, Parkinson's disease, and various cancers. The gene
delivery system is mainly categorized to be viral and nonviral vectors. Viral
vectors are biological systems derived from naturally evolved viruses capable
of transferring their genetic material into the host cells, and thus are very
RSC Polymer Chemistry Series No. 3
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