Direct central nervous system delivery provides enhanced protection following vector mediated gene replacement in a severe model of Spinal Muscular Atrophy

Biochemical and Biophysical Research Communications

Volumn 417, Issue 1, 2012, Pages 376-381

  Glascock, Jacqueline J ^a,b   Shababi, Monir ^a   Wetz, Mary J ^a,b   Krogman, Megan M ^a,b   Lorson, Christian L ^a,b  

^a UNIVERSITY OF MISSOURI   (United States)

^b University of Missouri   (United States)

Author keywords

Gene therapy; Neurodegeneration; ScAAV; Spinal Muscular Atrophy (SMA); Survival Motor Neuron (SMN); Therapeutics

Indexed keywords

ADENOVIRUS VECTOR; COMPLEMENTARY DNA; SURVIVAL MOTOR NEURON PROTEIN;

ADENO ASSOCIATED VIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BODY WEIGHT; CONTROLLED STUDY; GENE REPLACEMENT THERAPY; GENETIC TRANSFECTION; LIFESPAN; MOUSE; NEWBORN; NONHUMAN; NUCLEOTIDE SEQUENCE; PRIORITY JOURNAL; SPINAL MUSCULAR ATROPHY; SURVIVAL; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY;

ANIMALS; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC COMPLEMENTATION TEST; GENETIC VECTORS; INJECTIONS, INTRAVENTRICULAR; MICE; MUSCULAR ATROPHY, SPINAL; SURVIVAL OF MOTOR NEURON 1 PROTEIN; SURVIVAL OF MOTOR NEURON 2 PROTEIN;

ADENO-ASSOCIATED VIRUS; ANIMALIA; MUS;

EID: 84855760635     PISSN: 0006291X     EISSN: 10902104     Source Type: Journal    
DOI: 10.1016/j.bbrc.2011.11.121     Document Type: Article

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