Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates - Meyer, Kathrin; Ferraiuolo, Laura; Schmelzer, Leah; Braun, Lyndsey; McGovern, Vicki; Likhite, Shibi; Michels, Olivia; Govoni, Alessandra; Fitzgerald, Julie; Morales, Pablo; Foust, Kevin D; Mendell, Jerry R; et al. (Molecular therapy, 2015)
Keywords: Animals; Animals, Newborn; Atrophy; Brain research; Brain Stem - metabolism; Cerebral Cortex - metabolism; Dependovirus - genetics; Disease Models, Animal; DNA, Complementary - administration & dosage; DNA, Complementary - genetics; DNA, Complementary - metabolism; Dose-Response Relationship, Drug; Gene Expression; Gene therapy; Genetic Therapy - methods; Genetic Vectors - administration & dosage; Genetic Vectors - pharmacokinetics; Injections, Epidural; Macaca fascicularis; Mice; Mice, Knockout; Motor Neurons - metabolism; Motor Neurons - pathology; Muscular Atrophy, Spinal - genetics; Muscular Atrophy, Spinal - metabolism; Muscular Atrophy, Spinal - pathology; Muscular Atrophy, Spinal - therapy; Neurons; Original; Proteins; Spinal cord; Spinal Cord - metabolism; Spinal Cord - pathology; Survival of Motor Neuron 1 Protein - genetics; Survival of Motor Neuron 1 Protein - metabolism; Transduction, Genetic; Transgenes; United States--US; Ohio