text: rAAV vector-mediated sarcogylcan gene transfer in a hamster model for limb girdle muscular dystrophy. - PubMed - NCBI
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  See comment in PubMed Commons belowGene Ther. 1999 Jan;6(1):74-82.rAAV vector-mediated sarcogylcan gene transfer in a hamster model for limb girdle muscular dystrophy.Li J1, Dressman D, Tsao YP, Sakamoto A, Hoffman EP, Xiao X.Author information1Department of Molecular Genetics and Biochemistry, University of Pittsburgh School of Medicine, PA 15261, USA.AbstractThe limb girdle muscular dystrophies (LGMD) are a genetically and phenotypically heterogeneous group of degenerative neuromuscular diseases. A subset of the genetically recessive forms of LGMD are caused by mutations in the four muscle sarcoglycan genes (alpha, beta, gamma and delta). The coding sequences of all known sarcoglycan genes are smaller than 2 kb, and thus can be readily packaged in recombinant adeno-associated virus (rAAV) vectors. Previously, we have demonstrated highly efficient and sustained transduction in mature muscle tissue of immunocompetent animals with rAAV vectors. In this report, we utilize recombinant AAV containing the delta-sarcoglycan gene for genetic complementation of muscle diseases using a delta-sarcoglycan-deficient hamster model (Bio 14.6). We show efficient delivery and widespread expression of delta-sarcoglycan after a single intramuscular injection. Importantly, rAAV vector containing the human delta-sarcoglycan cDNA restored secondary biochemical deficiencies, with correct localization of other sarcoglycan proteins to the muscle fiber membrane. Interestingly, restoration of alpha-, as well as beta-sarcoglycan was homogeneous and properly localized throughout transduced muscle, and appeared unaffected by dramatic overexpression of delta-sarcoglycan in the cytoplasm of some myofibers. These results support the feasibility of rAAV vector's application to treat LGMD by means of direct in vivo gene transfer.PMID: 10341878 DOI: 10.1038/sj.gt.3300830 [PubMed - indexed for MEDLINE] Free full textSharePublication Types, MeSH Terms, SubstancesPublication TypesResearch Support, Non-U.S. Gov'tMeSH TermsAnimalsBlotting, WesternCricetinaeCytoskeletal Proteins/genetics*Cytoskeletal Proteins/metabolismDependovirus/genetics*Gene ExpressionGene Transfer Techniques*Genetic Therapy/methods*Genetic Vectors/genetics*HumansInjections, IntramuscularMembrane Glycoproteins/genetics*Membrane Glycoproteins/metabolismMicroscopy, FluorescenceMuscle, Skeletal/metabolismMuscular Dystrophies/metabolismMuscular Dystrophies/therapy*RegenerationSarcoglycansSubstancesCytoskeletal ProteinsMembrane GlycoproteinsSarcoglycansLinkOut - more resourcesFull Text SourcesNature Publishing GroupOther Literature SourcesCOS Scholar UniverseCited by Patents in - The LensMedicalLimb-girdle muscular dystrophy - Genetic AllianceMuscular dystrophy - Genetic AllianceGenes and Gene Therapy - MedlinePlus Health InformationMuscular Dystrophy - MedlinePlus Health InformationPubMed Commons home