Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: A dose-response study in mice and nonhuman primates

Molecular Therapy

Volumn 23, Issue 3, 2015, Pages 477-487

  Meyer, Kathrin ^a   Ferraiuolo, Laura ^a   Schmelzer, Leah ^a   Braun, Lyndsey ^a   McGovern, Vicki ^b   Likhite, Shibi ^a,c   Michels, Olivia ^a   Govoni, Alessandra ^a   Fitzgerald, Julie ^c   Morales, Pablo ^d   Foust, Kevin D ^c   Mendell, Jerry R ^a,c   Burghes, Arthur H M ^b   Kaspar, Brian K ^a,c  

^a THE RESEARCH INSTITUTE AT NATIONWIDE CHILDREN S HOSPITAL   (United States)

^b THE OHIO STATE UNIVERSITY WEXNER MEDICAL CENTER   (United States)

^c OHIO STATE UNIVERSITY   (United States)

^d Haman Ranch   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BETA ACTIN; PARVOVIRUS VECTOR; COMPLEMENTARY DNA; SURVIVAL MOTOR NEURON PROTEIN 1;

ADENO ASSOCIATED VIRUS 9; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BRAIN; BRAIN STEM; CELL SURVIVAL; CEREBROSPINAL FLUID; DOSE RESPONSE; GENE EXPRESSION; GENE THERAPY; GENETIC TRANSDUCTION; INTERMETHOD COMPARISON; INTRACEREBROVENTRICULAR DRUG ADMINISTRATION; INTRAVENOUS DRUG ADMINISTRATION; MOTONEURON; MOUSE; MOUSE MODEL; NONHUMAN; PATIENT POSITIONING; PRIMATE; SPINAL MUSCULAR ATROPHY; TRANSGENE; ADMINISTRATION AND DOSAGE; ANIMAL; BRAIN CORTEX; DEPENDOPARVOVIRUS; DISEASE MODEL; EPIDURAL DRUG ADMINISTRATION; GENE VECTOR; GENETICS; KNOCKOUT MOUSE; MACACA FASCICULARIS; METABOLISM; MUSCULAR ATROPHY, SPINAL; NEWBORN; PATHOLOGY; PHARMACOKINETICS; PROCEDURES; SPINAL CORD;

ADENO-ASSOCIATED VIRUS 9; MUS; PRIMATES;

ANIMALS; ANIMALS, NEWBORN; BRAIN STEM; CEREBRAL CORTEX; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; DNA, COMPLEMENTARY; DOSE-RESPONSE RELATIONSHIP, DRUG; GENE EXPRESSION; GENETIC THERAPY; GENETIC VECTORS; INJECTIONS, EPIDURAL; MACACA FASCICULARIS; MICE; MICE, KNOCKOUT; MOTOR NEURONS; MUSCULAR ATROPHY, SPINAL; SPINAL CORD; SURVIVAL OF MOTOR NEURON 1 PROTEIN; TRANSDUCTION, GENETIC; TRANSGENES;

EID: 84924066675     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2014.210     Document Type: Article

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