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 t...

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Bibliographic Details
Published in:Journal of drug targeting 2005-02, Vol.13 (2), p.81-87
Main Authors: Weisbart, Richard H., Hansen, James E., Nishimura, Robert N., Chan, Grace, Wakelin, Rika, Chang, Sophia S., Baresi, Larry, Chamberlain, Jeffrey S.
Format: Article
Language:English
Subjects:
Animals
Antibody
Biological and medical sciences
Blotting, Western
Cell Line
Cell Membrane - metabolism
Cercopithecus aethiops
Cloning, Molecular
Cytoplasm - metabolism
Dystroglycans - metabolism
dystrophin
Dystrophin - genetics
Dystrophin - metabolism
General pharmacology
Humans
Immunoglobulin Fragments
Lymphokines
Medical sciences
muscular dystrophy
Myoblasts - metabolism
Pharmaceutical technology. Pharmaceutical industry
Pharmacology. Drug treatments
Pichia - metabolism
Pichia pastoris
Protein Sorting Signals
Protein Transport
Recombinant Fusion Proteins - genetics
Recombinant Fusion Proteins - metabolism
Sialoglycoproteins
Transfection
transport
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Summary: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.
ISSN:1061-186X
1029-2330
DOI:10.1080/10611860400029002