An intracellular delivery vehicle for protein transduction of micro-dystrophin.


The Fv fragment of an antibody that selectively targets and penetrates skeletal muscle in vivo was produced as a fusion protein with a micro-dystrophin for use as a delivery vehicle to transport micro-dystrophin into muscle cells. Fv-micro-dystrophin was produced as a secreted protein by transient transfection of Fv-micro-dystrophin cDNA in COS-7 cells and as a non-secreted protein by permanent transfection in Pichia pastoris. Isolated Fv-micro-dystrophin was shown to be full-length by Western blot analysis. Fv-micro-dystrophin penetrated multiple cell lines in vitro, and it localized to the plasma membrane of a cell line with membrane beta-dystroglycan. In the absence of membrane beta-dystroglycan, it localized to the cytoplasm. Antibody-mediated transduction of micro-dystrophin into muscle cells is a potential therapy for dystrophin-deficient muscular dystrophies.

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@article{Weisbart2005AnID, title={An intracellular delivery vehicle for protein transduction of micro-dystrophin.}, author={Richard H. Weisbart and James E. Hansen and Robert N. Nishimura and G. M. T. W. Chan and Rika Wakelin and Sophia S Chang and Larry Baresi and Jeffrey S. Chamberlain}, journal={Journal of drug targeting}, year={2005}, volume={13 2}, pages={81-7} }