Non-viral gene therapy for neurological diseases, with an emphasis on targeted gene delivery
作者： Mary-Louise Rogers , Robert Archer Rush
DOI: 10.1016/J.JCONREL.2011.08.026
关键词:
摘要: Non-viral gene therapy systems are considered safer than viral delivery. This article reviews recent research describing novel, non-viral delivery to the central nervous system, with a special emphasis on receptor mediated using antibodies (termed immunogenes) specific receptors. By targeting agents such as that can be retrogradely transported within neurons, therapies deliver genes neurons protected by blood brain barrier. Components of effective described including DNA/RNA carriers, receptor-mediated endocytosis, endosomal escape and nuclear entry. In addition, stealth polyethylene glycol used improve in-vivo discussed. The value immunogenes therapeutic for fatal diseases Amyotrophic Lateral Sclerosis is significant but further work confirm efficacy required before truly achieved.
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sci-hub.se 参考文章(110)
K. Stöckel, M. Schwab, H. Thoenen, Comparison between the retrograde axonal transport of nerve growth factor and tetanus toxin in motor, sensory and adrenergic neurons Brain Research. ,vol. 99, pp. 1- 16 ,(1975) , 10.1016/0006-8993(75)90604-6
E.J. Kwon, J.M. Bergen, I.K. Park, S.H. Pun, Peptide-modified vectors for nucleic acid delivery to neurons. Journal of Controlled Release. ,vol. 132, pp. 230- 235 ,(2008) , 10.1016/J.JCONREL.2008.06.012
Ayumi Sato, Motoki Takagi, Akira Shimamoto, Shigeru Kawakami, Mitsuru Hashida, Small interfering RNA delivery to the liver by intravenous administration of galactosylated cationic liposomes in mice. Biomaterials. ,vol. 28, pp. 1434- 1442 ,(2007) , 10.1016/J.BIOMATERIALS.2006.11.010
R Kircheis, A Kichler, G Wallner, M Kursa, M Ogris, T Felzmann, M Buchberger, E Wagner, Coupling of cell-binding ligands to polyethylenimine for targeted gene delivery Gene Therapy. ,vol. 4, pp. 409- 418 ,(1997) , 10.1038/SJ.GT.3300418
S BARATI, P HURTADO, S ZHANG, R TINSLEY, I FERGUSON, R RUSH, GDNF gene delivery via the p75NTR receptor rescues injured motor neurons Experimental Neurology. ,vol. 202, pp. 179- 188 ,(2006) , 10.1016/J.EXPNEUROL.2006.05.027
Nina E. Weisser, J. Christopher Hall, Applications of single-chain variable fragment antibodies in therapeutics and diagnostics Biotechnology Advances. ,vol. 27, pp. 502- 520 ,(2009) , 10.1016/J.BIOTECHADV.2009.04.004
M G Toscano, Z Romero, P Muñoz, M Cobo, K Benabdellah, F Martin, Physiological and tissue-specific vectors for treatment of inherited diseases. Gene Therapy. ,vol. 18, pp. 117- 127 ,(2011) , 10.1038/GT.2010.138
Erwei Song, Pengcheng Zhu, Sang-Kyung Lee, Dipanjan Chowdhury, Steven Kussman, Derek M Dykxhoorn, Yi Feng, Deborah Palliser, David B Weiner, Premlata Shankar, Wayne A Marasco, Judy Lieberman, Antibody mediated in vivo delivery of small interfering RNAs via cell-surface receptors Nature Biotechnology. ,vol. 23, pp. 709- 717 ,(2005) , 10.1038/NBT1101
Jiabing Chen, Shinobu Gamou, Atsushi Takayanagi, Nobuyoshi Shimizu, A novel gene delivery system using EGF receptor-mediated endocytosis FEBS Letters. ,vol. 338, pp. 167- 169 ,(1994) , 10.1016/0014-5793(94)80357-9
Yajun Duan, Junnian Zheng, Sufang Han, Yi Wu, Yanming Wang, Deguan Li, Deling Kong, Yaoting Yu, A tumor targeted gene vector modified with G250 monoclonal antibody for gene therapy. Journal of Controlled Release. ,vol. 127, pp. 173- 179 ,(2008) , 10.1016/J.JCONREL.2008.01.016