An intracellular delivery vehicle for protein transduction of micro-dystrophin
| Autor: | Grace Chan, Robert N. Nishimura, Sophia S. Chang, Richard H. Weisbart, Jeffrey S. Chamberlain, Rika Wakelin, James E. Hansen, Larry Baresi |
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| Rok vydání: | 2005 |
| Předmět: |
musculoskeletal diseases
congenital hereditary and neonatal diseases and abnormalities Vesicle-associated membrane protein 8 Cytoplasm Recombinant Fusion Proteins Sialoglycoproteins Blotting Western Pharmaceutical Science Protein Sorting Signals Transfection Pichia Cell Line Dystrophin Myoblasts Transduction (genetics) Chlorocebus aethiops medicine Myocyte Animals Humans Cloning Molecular Dystroglycans Immunoglobulin Fragments Lymphokines biology Cell Membrane Skeletal muscle musculoskeletal system Fusion protein Transport protein Cell biology Protein Transport medicine.anatomical_structure Biochemistry biology.protein |
| Zdroj: | Journal of drug targeting. 13(2) |
| ISSN: | 1061-186X |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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