Enhanced effect of microdystrophin gene transfection by HSV-VP22 mediated intercellular protein transport

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Enhanced effect of microdystrophin gene transfection by HSV-VP22 mediated intercellular protein transport

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dc.contributor.author Xiong, Fu en_US
dc.contributor.author Xiao, Shaobo en_US
dc.contributor.author Yu, Meijuan en_US
dc.contributor.author Li, Wanyi en_US
dc.contributor.author Zheng, Hui en_US
dc.contributor.author Shang, Yanchang en_US
dc.contributor.author Peng, Funing en_US
dc.contributor.author Zhao, Cuiping en_US
dc.contributor.author Zhou, Wenliang en_US
dc.contributor.author Chen, Huanchun en_US
dc.contributor.author Fang, Liurong en_US
dc.contributor.author Chamberlain, Jeffrey S. en_US
dc.contributor.author Zhang, Cheng en_US
dc.date.accessioned 2010-04-21T15:48:38Z
dc.date.available 2010-04-21T15:48:38Z
dc.date.issued 2007 en_US
dc.identifier.citation Xiong F, Xiao S, Yu M, et al. Enhanced effect of microdystrophin gene transfection by HSV-VP22 mediated intercellular protein transport. BMC Neuroscience. 2007;8(1):50. en_US
dc.identifier.other 10.1186/1471-2202-8-50 en_US
dc.identifier.uri http://www.biomedcentral.com/1471-2202/8/50 en_US
dc.identifier.uri http://hdl.handle.net/1773/15717
dc.description.abstract Background: Duchenne musclar dystrophy (DMD) is an X-linked recessive disease caused by mutations of dystrophin gene, there is no effective treatment for this disorder at present. Plasmidmediated gene therapy is a promising therapeutical approach for the treatment of DMD. One of the major issues with plasmid-mediated gene therapy for DMD is poor transfection efficiency and distribution. The herpes simplex virus protein VP22 has the capacity to spread from a primary transduced cell to surrounding cells and improve the outcome of gene transfer. To improve the efficiency of plasmid-mediated gene therapy and investigate the utility of the intercellular trafficking properties of VP22-linked protein for the treatment for DMD, expression vectors for C-terminal versions of VP22-microdystrophin fusion protein was constructed and the VP22-mediated shuttle effect was evaluated both in vitro and in vivo. Results: Our results clearly demonstrate that the VP22-microdystrophin fusion protein could transport into C2C12 cells from 3T3 cells, moreover, the VP22-microdystrophin fusion protein enhanced greatly the amount of microdystrophin that accumulated following microdystrophin gene transfer in both transfected 3T3 cells and in the muscles of dystrophin-deficient (mdx) mice. Conclusion: These results highlight the efficiency of the VP22-mediated intercellular protein delivery for potential therapy of DMD and suggested that protein transduction may be a potential and versatile tool to enhance the effects of gene delivery for somatic gene therapy of DMD. en_US
dc.description.sponsorship National Natural Science Foundation of China (30370510, 30170337); CMB Fund (4209347); the Key Project of the State Ministry of Public Health (2001321); and National Nature Science Foundation of China (30400322). en_US
dc.language.iso en_US en_US
dc.title Enhanced effect of microdystrophin gene transfection by HSV-VP22 mediated intercellular protein transport en_US
dc.type Article en_US


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