Sustained expression and safety of human GNE in normal mice after gene transfer based on AAV8 systemic delivery

Keyphrases

• Gene Transfer 100%
• Sustained Expression 100%
• Systemic Delivery 100%
• Transfer Based 100%
• AAV8 100%
• GNE Myopathy 100%
• Muscle Cells 66%
• Viral Vectors 66%
• GNE mutations 66%
• Gene Therapy 33%
• Overexpression 33%
• Cells in Culture 33%
• Autosomal Recessive 33%
• Complementary DNA (cDNA) 33%
• Acetylation 33%
• Myopathy 33%
• Virus-like Particles 33%
• Adult-onset 33%
• Skeletal muscle 33%
• Intravenous Administration 33%
• Biosynthesis Pathway 33%
• Sialic Acid 33%
• Luciferase 33%
• Therapy Approaches 33%
• Bifunctional Enzyme 33%
• General Toxicity 33%
• GNE Gene 33%
• UDP-N-acetylglucosamine Pyrophosphorylase 33%
• Mannosamine 33%
• Healthy Mice 33%

Biochemistry, Genetics and Molecular Biology

• Enzyme 100%
• Wild Type 100%
• Transgene 100%
• Virus Vector 100%
• Stable Expression 100%
• Gene Therapy 50%
• Phosphotransferase 50%
• Kinase 50%
• Luciferase 50%
• Anabolism 50%
• Skeletal Muscle 50%
• N-Acetylglucosamine 50%
• Autosomal Recessive Inheritance 50%
• Uridine Diphosphate 50%
• 2-Epimerase 50%
• GNE (Gene) 50%
• Virus Particle 50%
• Messenger RNA 50%
• Sialic Acid 50%
• Mannosamine 50%