Chitosan and its modifications: are they possible vehicles for gene therapy

Kedjarune-Leggat, Ureporn, and Leggat, Peter A. (2011) Chitosan and its modifications: are they possible vehicles for gene therapy. In: Yuan, Xu-bo, (ed.) Non-Viral Gene Therapy. InTech, Rijeka, Croatia, pp. 439-454.

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[Extract] In gene therapy, the most important step is how to effectively deliver the therapeutic gene to the target cells or organ. At present, there are two methods, which are those using a viral and a non-viral vector system. The most common viral vectors that have been used include retroviruses, herpes simplex viruses, lentiviruses, adenoviruses and adeno-associated viruses (Oligino et al., 2000). The advantages of most viral vectors are high transfection efficiency and fast transcription of the foreign material inserted in the viral genome. However, a number of disadvantages have also been described, such as poor target-specificity, low capacity to incorporate foreign deoxyribonucleic acid (DNA) sequence to their genome (Mansouri et al., 2004), toxic and inflammatory effects, wild– type mutations, potential oncogenic effects (Lee et al., 1998), and, in particular, unwanted immune responses. In clinical trials of gene replacement therapy using viral vectors, significant adverse effects have been reported, including a fatal inflammatory response associated with adenoviral vector (Raper et al., 2003), and the development of acute leukaemia in recipients of ex-vivo, adenoviral vector-transduced hematopoietic cells (Woods et al., 2006). Intravenous adenoviral vector has also resulted in high liver toxicity due to uptake by hepatocytes or Kupffer cells of the liver reticular endothelial system, immediately following systemic administration.

Item ID: 21397
Item Type: Book Chapter (Research - B1)
ISBN: 978-953-207-538-9
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© 2011 InTech. All chapters are Open Access distributed under the Creative Commons Attribution 3.0 license, which permits to copy, distribute, transmit, and adapt the work in any medium, so long as the original work is properly cited.

Date Deposited: 11 Apr 2012 04:39
FoR Codes: 11 MEDICAL AND HEALTH SCIENCES > 1117 Public Health and Health Services > 111706 Epidemiology @ 100%
SEO Codes: 92 HEALTH > 9201 Clinical Health (Organs, Diseases and Abnormal Conditions) > 920113 Oro-Dental Disorders @ 100%
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