Phase I clinical trial utilizing gene therapy for limb girdle muscular dystrophy: alpha-, beta-, gamma-, or delta-sarcoglycan gene delivered with intramuscular instillations of adeno-associated vectors

MeSH terms

• Adenoviridae / genetics
• Animals
• Clinical Protocols
• Clinical Trials, Phase I as Topic
• Cytoskeletal Proteins / genetics*
• Cytoskeletal Proteins / pharmacology
• Dystroglycans
• Gene Transfer Techniques
• Genetic Therapy / methods*
• Genetic Vectors / administration & dosage*
• Genetic Vectors / genetics
• Humans
• Injections, Intramuscular
• Membrane Glycoproteins / genetics*
• Membrane Glycoproteins / pharmacology
• Mice
• Mice, Knockout
• Muscular Dystrophies / genetics
• Muscular Dystrophies / therapy*
• Sarcoglycans