Functional Nanoparticles for Molecular Imaging-Guided Gene Delivery and Therapy

The inefficient delivery of genes to target tissues and the inability to monitor delivery of genes and therapeutic responses at both cellular and tissue level hinder the success of gene therapy. Fortunately, molecular imaging strategy provides a vital rol

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Functional Nanoparticles for Molecular Imaging-Guided Gene Delivery and Therapy Tianxin Miao, Yu Zhang, Yun Zeng, Rui Tian, and Gang Liu

8.1

Introduction

Gene therapy has been tremendously applied in treatment of various diseases and disorders. Over 1800 gene therapy clinical trials have been completed, ongoing, or already gained approval worldwide until the year 2012, providing an effective and global recognized effect treatment for cancer, cardiovascular, and other severe diseases [1]. However, the inefficient delivery of genes to target tissues and the inability to monitor delivery of genes and therapeutic responses at both cellular and tissue level hinder the success of gene therapy. Fortunately, molecular imaging strategy provides a vital role in assisting gene therapy in a noninvasive and spatiotemporal manner. To better optimize the effectiveness of gene therapy, numerous functional

T. Miao Bioengineering Program, College of Engineering and Mathematical Sciences, College of Medicine, University of Vermont, Burlington, VT 05405, USA Y. Zhang State Key Laboratory of Bioelectronics, Jiangsu Key Laboratory for Biomaterials and Devices, School of Biological Science and Medical Engineering, Southeast University, Nanjing 210096, People’s Republic of China Y. Zeng State Key Laboratory of Molecular Vaccinology and Molecular Diagnostics and Center for Molecular Imaging and Translational Medicine, School of Public Health, Xiamen University, Xiamen, 361102, China Department of Pharmacology, West China School of Preclinical and Forensic Medicine, Sichuan University, Chengdu, 610041, China R. Tian • G. Liu (*) State Key Laboratory of Molecular Vaccinology and Molecular Diagnostics and Center for Molecular Imaging and Translational Medicine, School of Public Health, Xiamen University, Xiamen, 361102, China e-mail: [email protected] © Springer Science+Business Media Singapore 2016 Z. Dai (ed.), Advances in Nanotheranostics II, Springer Series in Biomaterials Science and Engineering 7, DOI 10.1007/978-981-10-0063-8_8

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Fig. 8.1 Schematic representation of multifunctional nanoparticles for molecular imaging-guided gene delivery (Reprinted from Ref. [2], Copyright 2010, with permission from Elsevier)

nanoparticles have been developed to achieve this goal while visualizing the delivery process (Fig. 8.1) [2]. This chapter will provide an overview of various functionalized nanoparticles with unique physiochemical properties for molecular imaging-guided gene therapy. In addition, the concept of functionality and cell response will be explored. These multifunctional systems will promote diagnostic evaluation and gene therapy development and predict clinical outcomes, achieving the promise of personalized and advanced medicine. Gene therapy has great potential to treat various diseases ranging from cystic fibrosis, macular degeneration, Parkinson’s disease, and different types of cancers [3–6]. The definition of gene therapy is transferring therapeutic genes into a cell to replace or silence de