title: Chemical modification of chitosan for efficient gene therapy.
abstract: Gene therapy involves the introduction of foreign genetic material into cells in order to exert a therapeutic effect. Successful gene therapy relies on effective vector system. Viral vectors are highly efficient in transfecting cells, but the undesirable complications limit their therapeutic applications. As a natural biopolymer, chitosan has been considered to be a good gene carrier candidate due to its ideal character which combines biocompatibility, low toxicity with high cationic density together. However, the low cell specificity and low transfection efficiency of chitosan as a gene carrier need to be overcome before undertaking clinical trials. This chapter is principally on those endeavors such as chemical modifications using cell-specific ligands and stimuli-response groups as well as penetrating modifications that have been done to increase the performances of chitosan in gene therapy.
authors.affiliation: Department of Pharmaceutics, State Key Laboratory of Natural Medicines, China Pharmaceutical University, Nanjing, PR China.
authors.forename: Hu-Lin
authors.initials: HL
authors.lastname: Jiang
authors.affiliation: School of Pharmacy, China Pharmaceutical University, Nanjing, PR China.
authors.forename: Peng-Fei
authors.initials: PF
authors.lastname: Cui
authors.affiliation: School of Pharmacy, China Pharmaceutical University, Nanjing, PR China.
authors.forename: Rong-Lin
authors.initials: RL
authors.lastname: Xie
authors.affiliation: Research Institute for Agriculture and Life Sciences, Seoul National University, Seoul, South Korea. Electronic address: chocs@plaza.snu.ac.kr.
authors.forename: Chong-Su
authors.initials: CS
authors.lastname: Cho
mesh_terms: D000596:Amino Acids / Q000737:chemistry; D000818:Animals; D001812:Blood-Brain Barrier; D048271:Chitosan / Q000737:chemistry*; D005492:Folic Acid / Q000737:chemistry; D005690:Galactose / Q000737:chemistry; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006801:Humans; D006820:Hyaluronic Acid / Q000737:chemistry; D006863:Hydrogen-Ion Concentration; D008024:Ligands; D008280:Magnetics; D008358:Mannose / Q000737:chemistry; D013438:Sulfhydryl Compounds / Q000737:chemistry; D014162:Transfection
chemical_list: D000596:Amino Acids; D008024:Ligands; D013438:Sulfhydryl Compounds; D006820:Hyaluronic Acid; D048271:Chitosan; D005492:Folic Acid; D008358:Mannose; D005690:Galactose
keywords: Chitosan; Gene therapy; Ligand modification; Stimuli response