In vivo gene therapy

Maruyama-Tabata FI, et al. (2000). Effective suicide gene therapy in vivo by EBV-based plasmid vector coupled with polyamidoamine dendrimer. Gene Ther. 7 53-60. Wang Y, et al. (2001). Combination of electroporation and DNA/dendrimer complexes enhances gene transfer into murine cardiac transplants. Am. J. Transplant. 1 334-338. 

The effect of serum on transfection efficiency is very important for the practical applications of gene therapy in vivo because transfection efficiency in vivo is inhibited by serum (Goldman et al. 1997). Sato et al. (2001) studied the effect of serum on the transfection efficiency of the complexes in vitro. The results showed that the transfection efficiency of the complexes was increased at np to 20% serum due to the increased cell function by the serum, and was decreased at 50% serum owing to cell damage, although PEl-mediated transfection was decreased at 10% semm (Erbacher et al. 1998). 

Maruyama-Tabata, H. et al.. Effective suicide gene therapy in vivo by EBV-based plasmid vector coupled with polyamidoamine dendrimer. Gene Ther., 7, 53,2000. 

Xing, J., et al., Polycationic nanoparticles as nonviral vectors employed for gene therapy in vivo. Mini Reviews in Medical Chemistry, 10 126-137,2010. 

Bookstein R, Demers W, Gregory R, Maneval D, Park J and Wills K (1996). p53 gene therapy in vivo of hepatocellular and liver metastatic colorectal cancer. Seminars in Oncology, 23(1), 66-77. 

Garver RI, Chytil A, Courtney M, Crystal RG. Clonal gene therapy in vivo expression of a transplanted monoclonal population of murine fibroblasts containing a retroviras inserted human alpha 1-antitiypsin gene. Trans Assoc Am Physicians 1987 100 10-20. 

Chen, S.H., Shine, H.D., Goodman, J.C., Grossman, R.G., Woo, S.L. (1994). Gene therapy for brain tumors regression of experimental ghomas by adenovirus-mediated gene transfer in vivo. Proc. Natl. Acad. Sci. U.S.A., 91(8), 3054-3057. 

Finegold, A.A., Mannes, A.J., ladarola, M.J. A paracrine paradigm for in vivo gene therapy in the central nervous system treatment of chronic pain, Hum. GeneTher. 1999, 10, 1251-1257. 

Lasic, D.D. (1997a) Recent developments in medical applications of liposomes sterically stabilized liposomes in cancer therapy and gene delivery in vivo. J. Controlled Release, 48,203-222. 

T. Tanaka, F. Kanai, K. H. Lan, M. Ohashi, Y. Shiratori, Y. Yoshida, H. Hamada, and M. Omata, Adenovirus-mediated gene therapy of gastric carcinoma using cancer-specific gene expression in vivo, Biochem. Biophys. Res. Commun. 231 115 (1997). 

Strategies applied to gene therapy. Ex vivo - the target cells are treated with the gene product outside the individual, expanded by cell culture, and then reintroduced into the patient or in vivo - the genetic material is introduced directly into the individual by a systemic route, or, whenever possible, site-specifically. 

Narfstrom K, Katz ML, Ford M, Redmond TM, Rakoczy E, Bragadottir R. In vivo gene therapy in young and adult RPE65 dogs produces long-term visual improvement. J Hered 2003 94 31-37. 

Bonadio J, et al. (1999). Localized, direct plasmid gene delivery in vivo prolonged therapy results in reproducible tissue regeneration. Nat. Med. 5 753-759. 

---