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http://purl.uniprot.org/citations/12054513http://www.w3.org/1999/02/22-rdf-syntax-ns#typehttp://purl.uniprot.org/core/Journal_Citation
http://purl.uniprot.org/citations/12054513http://www.w3.org/2000/01/rdf-schema#comment"The adeno-associated virus vector is a good tool for gene transfer into skeletal muscle, but the length of a gene that can be incorporated is limited. To develop a gene therapy for Duchenne muscular dystrophy, we generated a series of rod-truncated micro-dystrophin cDNAs: M3 (one rod repeat, 3.9 kb), AX11 (three rod repeats, 4.4 kb), and CS1 (four rod repeats, 4.9 kb). These micro-dystrophins, driven by a CAG promoter, were used to produce transgenic (Tg) mdx mice and all three micro-dystrophins were shown to localize at the sarcolemma together with the expression of dystrophin-associated proteins. Among them, CS1 greatly improved dystrophic phenotypes of mdx mice and contractile force of the diaphragm in particular was restored to the level of normal C57BL/10 mice. AX11 modestly ameliorated the dystrophic pathology, but, importantly, M3-Tg mdx mice still showed severe dystrophic phenotypes. These data suggest that the rod structure, and its length in particular, is crucial for the function of micro-dystrophin."xsd:string
http://purl.uniprot.org/citations/12054513http://purl.org/dc/terms/identifier"doi:10.1016/s0006-291x(02)00362-5"xsd:string
http://purl.uniprot.org/citations/12054513http://purl.uniprot.org/core/author"Suzuki M."xsd:string
http://purl.uniprot.org/citations/12054513http://purl.uniprot.org/core/author"Takeda S."xsd:string
http://purl.uniprot.org/citations/12054513http://purl.uniprot.org/core/author"Yoshimura M."xsd:string
http://purl.uniprot.org/citations/12054513http://purl.uniprot.org/core/author"Yuasa K."xsd:string
http://purl.uniprot.org/citations/12054513http://purl.uniprot.org/core/author"Sakamoto M."xsd:string
http://purl.uniprot.org/citations/12054513http://purl.uniprot.org/core/author"Yokota T."xsd:string
http://purl.uniprot.org/citations/12054513http://purl.uniprot.org/core/author"Ikemoto T."xsd:string
http://purl.uniprot.org/citations/12054513http://purl.uniprot.org/core/author"Miyagoe-Suzuki Y."xsd:string
http://purl.uniprot.org/citations/12054513http://purl.uniprot.org/core/author"Dickson G."xsd:string
http://purl.uniprot.org/citations/12054513http://purl.uniprot.org/core/date"2002"xsd:gYear
http://purl.uniprot.org/citations/12054513http://purl.uniprot.org/core/name"Biochem Biophys Res Commun"xsd:string
http://purl.uniprot.org/citations/12054513http://purl.uniprot.org/core/pages"1265-1272"xsd:string
http://purl.uniprot.org/citations/12054513http://purl.uniprot.org/core/title"Micro-dystrophin cDNA ameliorates dystrophic phenotypes when introduced into mdx mice as a transgene."xsd:string
http://purl.uniprot.org/citations/12054513http://purl.uniprot.org/core/volume"293"xsd:string
http://purl.uniprot.org/citations/12054513http://www.w3.org/2004/02/skos/core#exactMatchhttp://purl.uniprot.org/pubmed/12054513
http://purl.uniprot.org/citations/12054513http://xmlns.com/foaf/0.1/primaryTopicOfhttps://pubmed.ncbi.nlm.nih.gov/12054513
http://purl.uniprot.org/uniprot/#_A0A023ZT56-mappedCitation-12054513http://www.w3.org/1999/02/22-rdf-syntax-ns#objecthttp://purl.uniprot.org/citations/12054513
http://purl.uniprot.org/uniprot/#_A0A023ZTV5-mappedCitation-12054513http://www.w3.org/1999/02/22-rdf-syntax-ns#objecthttp://purl.uniprot.org/citations/12054513
http://purl.uniprot.org/uniprot/#_A0A1Q1NN66-mappedCitation-12054513http://www.w3.org/1999/02/22-rdf-syntax-ns#objecthttp://purl.uniprot.org/citations/12054513
http://purl.uniprot.org/uniprot/#_A0A1Q1PRS2-mappedCitation-12054513http://www.w3.org/1999/02/22-rdf-syntax-ns#objecthttp://purl.uniprot.org/citations/12054513
http://purl.uniprot.org/uniprot/#_A0A1Q1PRS4-mappedCitation-12054513http://www.w3.org/1999/02/22-rdf-syntax-ns#objecthttp://purl.uniprot.org/citations/12054513
http://purl.uniprot.org/uniprot/#_A0A1Q1PRS6-mappedCitation-12054513http://www.w3.org/1999/02/22-rdf-syntax-ns#objecthttp://purl.uniprot.org/citations/12054513