Non-viral Gene Therapy - Intracellular Delivery: Fundamentals and Applications - page 599

Non-viral Gene Therapy
Jianxiang Zhang, Xiaohui Li, Liping Lou, Xiaodong Li,
Yi Jia, Zhe Jin, and Yuxuan Zhu
Abstract Gene therapy has been considered to be a powerful approach for the
prevention and/or treatment of a variety of diseases from genetic disorders,
infections, to cancer. The success of gene therapy in the clinic is largely limited
currently, mainly due to the lack of safe and efficient delivery vectors. Despite the
high transfection efficiency, viral vectors encounter the vital toxicity issues and
production problems. Increasing endeavors have been therefore directed towards
the development of non-viral systems with the advantages of low immunogenicity
and toxicity, ease in manufacturing and mass production, low cost, excellent stability,
reduced vector size limitations, high flexibility regarding the size of transgenes to
be delivered, and diverse chemical designs for constructing vectors with multiple
functions. In this chapter, we summarized most of the synthetic non-viral systems
currently employed for gene therapy, including lipid and polymer-based vectors,
nanomaterials such as magnetic nanoparticles, quantum dots, gold/silica nano-
structures, carbon nanotubes, calcium phosphate nanoparticles, and layered double
hydroxides/clays, as well as multifunctional nanosystems among them. Particular
attention has been paid on synthetic polymers and the related nanomedicines.
Selected clinical trials of gene therapy using non-viral vectors as well as the future
development in this rapidly growing field were briefly discussed.
J. Zhang ( * ), X. Li ( * ), Y. Jia, and Y. Zhu
Department of Pharmaceutics, College of Pharmacy, Third Military Medical University,
30 Gaotanyan Zhengjie, Shapingba District, Chongqing 400038, China
e-mail: [e-mail]; [e-mail]
Z. Jin
Company 13, Brigade of Cadets, Third Military Medical University,
30 Gaotanyan Zhengjie, Shapingba District, Chongqing 400038, China
L. Lou
Polytechnic Institute of NYU, 6 MetroTech Center, Brooklyn, NY 11201, USA
X. Li
Department of Oral and Maxillofacial Surgery, The Affiliated Stomatology Hospital,
College of Medicine, Zhejiang University, Hangzhou 310006, Zhejiang, China