| scholarly article | Q13442814 |
| P50 | author | Marc Bartoli | Q42429987 |
| P2093 | author name string | Olivier Danos | |
| Isabelle Richard | |||
| Ludovic Arandel | |||
| Jérôme Poupiot | |||
| Evelyne Gicquel | |||
| Muriel Durand | |||
| Francoise Fougerousse | |||
| Nicolas Guerchet | |||
| P2860 | cites work | Missense mutations in the adhalin gene linked to autosomal recessive muscular dystrophy | Q24317764 |
| Filamin 2 (FLN2): A muscle-specific sarcoglycan interacting protein | Q24632485 | ||
| Sarcoglycan isoforms in skeletal muscle | Q28141128 | ||
| rAAV vector-mediated sarcogylcan gene transfer in a hamster model for limb girdle muscular dystrophy | Q28144597 | ||
| Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response | Q29619095 | ||
| Synthetic muscle promoters: activities exceeding naturally occurring regulatory sequences | Q30657785 | ||
| Muscle-specific promoters may be necessary for adeno-associated virus-mediated gene transfer in the treatment of muscular dystrophies. | Q31934596 | ||
| Full functional rescue of a complete muscle (TA) in dystrophic hamsters by adeno-associated virus vector-directed gene therapy | Q33793572 | ||
| AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. | Q34168942 | ||
| Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model | Q35847459 | ||
| Molecular and cell biology of the sarcoglycan complex | Q36152977 | ||
| Progressive muscular dystrophy in alpha-sarcoglycan-deficient mice | Q36276730 | ||
| Therapy insight: cardiovascular complications associated with muscular dystrophies | Q36304335 | ||
| Gene therapy strategies for Duchenne muscular dystrophy utilizing recombinant adeno-associated virus vectors. | Q36345325 | ||
| Further observations on the pathological responses of rat skeletal muscle to toxins isolated from the venom of the Australian tiger snake, Notechis scutatus scutatus | Q39868650 | ||
| MUSEAP, a novel reporter gene for the study of long-term gene expression in immunocompetent mice | Q40766588 | ||
| Rescue of skeletal muscles of gamma-sarcoglycan-deficient mice with adeno-associated virus-mediated gene transfer. | Q40863195 | ||
| Expression of the primary coxsackie and adenovirus receptor is downregulated during skeletal muscle maturation and limits the efficacy of adenovirus-mediated gene delivery to muscle cells | Q40957513 | ||
| Bidirectional signaling between sarcoglycans and the integrin adhesion system in cultured L6 myocytes | Q41070010 | ||
| Delivery of alpha- and beta-sarcoglycan by recombinant adeno-associated virus: efficient rescue of muscle, but differential toxicity | Q43655076 | ||
| Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors | Q43716886 | ||
| Long-term pharmacologically regulated expression of erythropoietin in primates following AAV-mediated gene transfer. | Q43727676 | ||
| Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector | Q43783192 | ||
| Early adenovirus-mediated gene transfer effectively prevents muscular dystrophy in alpha-sarcoglycan-deficient mice | Q43883312 | ||
| Repeated adeno-associated virus serotype 2 aerosol-mediated cystic fibrosis transmembrane regulator gene transfer to the lungs of patients with cystic fibrosis: a multicenter, double-blind, placebo-controlled trial | Q45693906 | ||
| Transduction and utility of the granulocyte-macrophage colony-stimulating factor gene into monocytes and dendritic cells by adeno-associated virus. | Q45744533 | ||
| Noninvasive monitoring of therapeutic gene transfer in animal models of muscular dystrophies. | Q45886068 | ||
| Safety and efficacy of AAV-mediated calpain 3 gene transfer in a mouse model of limb-girdle muscular dystrophy type 2A. | Q45888604 | ||
| Primary adhalinopathy (alpha-sarcoglycanopathy): clinical, pathologic, and genetic correlation in 20 patients with autosomal recessive muscular dystrophy. | Q46493735 | ||
| Functional rescue of the sarcoglycan complex in the BIO 14.6 hamster using delta-sarcoglycan gene transfer | Q47858156 | ||
| Force impairment in calpain 3-deficient mice is not correlated with mechanical disruption. | Q52550279 | ||
| Deficiency of a glycoprotein component of the dystrophin complex in dystrophic muscle | Q59089242 | ||
| A noninvasive procedure to detect muscle weakness in the mdx mouse | Q68845635 | ||
| Novel approach to quantitative polymerase chain reaction using real-time detection: application to the detection of gene amplification in breast cancer | Q77531704 | ||
| Femoral intra-arterial injection: a tool to deliver and assess recombinant AAV constructs in rodents whole hind limb | Q81364860 | ||
| Muscles of mice deficient in alpha-sarcoglycan maintain large masses and near control force values throughout the life span | Q81734671 | ||
| P433 | issue | 1 | |
| P304 | page(s) | 53-61 | |
| P577 | publication date | 2007-01-01 | |
| P1433 | published in | Molecular Therapy | Q15762400 |
| P1476 | title | Phenotypic correction of alpha-sarcoglycan deficiency by intra-arterial injection of a muscle-specific serotype 1 rAAV vector | |
| Phenotypic Correction of α-Sarcoglycan Deficiency by Intra-arterial Injection of a Muscle-specific Serotype 1 rAAV Vector. | |||
| P478 | volume | 15 |
| Q28611318 | 204th ENMC International Workshop on Biomarkers in Duchenne Muscular Dystrophy 24-26 January 2014, Naarden, The Netherlands |
| Q41823092 | A common disease-associated missense mutation in alpha-sarcoglycan fails to cause muscular dystrophy in mice |
| Q34814931 | A dystrophic muscle broadens the contribution and activation of immune cells reacting to rAAV gene transfer |
| Q36151226 | A translational approach for limb vascular delivery of the micro-dystrophin gene without high volume or high pressure for treatment of Duchenne muscular dystrophy |
| Q90732125 | AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice |
| Q37687423 | AAV-directed muscular dystrophy gene therapy |
| Q45866351 | AAV-mediated delivery of a mutated myostatin propeptide ameliorates calpain 3 but not alpha-sarcoglycan deficiency |
| Q28508977 | AAV-mediated intramuscular delivery of myotubularin corrects the myotubular myopathy phenotype in targeted murine muscle and suggests a function in plasma membrane homeostasis |
| Q38089518 | Animal models for metabolic, neuromuscular and ophthalmological rare diseases. |
| Q37944530 | Cell-matrix interactions in muscle disease |
| Q42209543 | Circulating miRNAs are generic and versatile therapeutic monitoring biomarkers in muscular dystrophies |
| Q57973234 | Eccentric contractions lead to myofibrillar dysfunction in muscular dystrophy |
| Q34566454 | Gene replacement therapies for duchenne muscular dystrophy using adeno-associated viral vectors |
| Q36014088 | Integration frequency and intermolecular recombination of rAAV vectors in non-human primate skeletal muscle and liver |
| Q35014916 | Interventions for muscular dystrophy: molecular medicines entering the clinic |
| Q35399100 | Intrinsic transgene immunogenicity gears CD8(+) T-cell priming after rAAV-mediated muscle gene transfer. |
| Q43268180 | Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins |
| Q40109350 | Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy. |
| Q45869725 | Long-term skeletal muscle protection after gene transfer in a mouse model of LGMD-2D. |
| Q34746653 | Mannosidase I inhibition rescues the human alpha-sarcoglycan R77C recurrent mutation |
| Q37707034 | Mesenchymal stem cells as therapeutic tools and gene carriers in liver fibrosis and hepatocellular carcinoma. |
| Q59358387 | Molecular Therapies for Muscular Dystrophies |
| Q35662071 | Myofiber Damage Evaluation by Evans Blue Dye Injection |
| Q92344995 | Plasmid-Mediated Gene Therapy in Mouse Models of Limb Girdle Muscular Dystrophy |
| Q36951286 | Precise Correction of Disease Mutations in Induced Pluripotent Stem Cells Derived From Patients With Limb Girdle Muscular Dystrophy |
| Q36761839 | Recent developments in the treatment of Duchenne muscular dystrophy and spinal muscular atrophy |
| Q36947058 | Relative persistence of AAV serotype 1 vector genomes in dystrophic muscle |
| Q35758335 | Sarcoglycanopathies: molecular pathogenesis and therapeutic prospects |
| Q36756695 | Serotype-specific Binding Properties and Nanoparticle Characteristics Contribute to the Immunogenicity of rAAV1 Vectors |
| Q35924068 | Serum proteomic profiling reveals fragments of MYOM3 as potential biomarkers for monitoring the outcome of therapeutic interventions in muscular dystrophies. |
| Q38908933 | Systemic delivery of adeno-associated viral vectors |
| Q38539938 | The Skeletal Muscle Environment and Its Role in Immunity and Tolerance to AAV Vector-Mediated Gene Transfer |
| Q37076027 | Vascular Regeneration in Ischemic Hindlimb by Adeno-Associated Virus Expressing Conditionally Silenced Vascular Endothelial Growth Factor |
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