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rdf:type |
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lifeskim:mentions |
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pubmed:issue |
2
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pubmed:dateCreated |
2005-4-12
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pubmed:abstractText |
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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pubmed:language |
eng
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pubmed:journal |
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pubmed:citationSubset |
IM
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pubmed:chemical |
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pubmed:status |
MEDLINE
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pubmed:month |
Feb
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pubmed:issn |
1061-186X
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pubmed:author |
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pubmed:issnType |
Print
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pubmed:volume |
13
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pubmed:owner |
NLM
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pubmed:authorsComplete |
Y
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pubmed:pagination |
81-7
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pubmed:meshHeading |
pubmed-meshheading:15823959-Animals,
pubmed-meshheading:15823959-Blotting, Western,
pubmed-meshheading:15823959-Cell Line,
pubmed-meshheading:15823959-Cell Membrane,
pubmed-meshheading:15823959-Cercopithecus aethiops,
pubmed-meshheading:15823959-Cloning, Molecular,
pubmed-meshheading:15823959-Cytoplasm,
pubmed-meshheading:15823959-Dystroglycans,
pubmed-meshheading:15823959-Dystrophin,
pubmed-meshheading:15823959-Humans,
pubmed-meshheading:15823959-Immunoglobulin Fragments,
pubmed-meshheading:15823959-Lymphokines,
pubmed-meshheading:15823959-Myoblasts,
pubmed-meshheading:15823959-Pichia,
pubmed-meshheading:15823959-Protein Sorting Signals,
pubmed-meshheading:15823959-Protein Transport,
pubmed-meshheading:15823959-Recombinant Fusion Proteins,
pubmed-meshheading:15823959-Sialoglycoproteins,
pubmed-meshheading:15823959-Transfection
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pubmed:year |
2005
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pubmed:articleTitle |
An intracellular delivery vehicle for protein transduction of micro-dystrophin.
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pubmed:affiliation |
Department of Research, Veterans Affairs Greater Los Angeles Healthcare System, Sepulveda, CA 91343, USA. rweisbar@ucla.edu
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pubmed:publicationType |
Journal Article
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