CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy

Journal of Clinical Investigation

Volumn 120, Issue 4, 2010, Pages 1253-1264

  Passini, Marco A ^a   Bu, Jie ^a   Roskelley, Eric M ^a   Richards, Amy M ^a   Sardi, S Pablo ^a   O'Riordan, Catherine R ^a   Klinger, Katherine W ^a   Shihabuddin, Lamya S ^a   Cheng, Seng H ^a  

^a GENZYME CORPORATION   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

PARVOVIRUS VECTOR; SURVIVAL MOTOR NEURON PROTEIN;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CENTRAL NERVOUS SYSTEM; CONTROLLED STUDY; DRUG EFFICACY; GENE THERAPY; LOCOMOTION; MOTOR COORDINATION; MOTOR PERFORMANCE; MOUSE; MUSCLE STRENGTH; NEUROMUSCULAR SYNAPSE; NONHUMAN; NUCLEOTIDE SEQUENCE; PRIORITY JOURNAL; PROTEIN EXPRESSION; SPINAL MUSCULAR ATROPHY; SURVIVAL RATE; VIRAL GENE DELIVERY SYSTEM;

ANIMALS; DISEASE MODELS, ANIMAL; GENE THERAPY; HUMANS; MICE; MOTOR NEURONS; MUSCLE STRENGTH; MUSCLE, SKELETAL; MUSCULAR ATROPHY, SPINAL; NEURITES; NEUROMUSCULAR JUNCTION; SURVIVAL OF MOTOR NEURON 1 PROTEIN;

EID: 77951201412     PISSN: 00219738     EISSN: 15588238     Source Type: Journal    
DOI: 10.1172/JCI41615     Document Type: Article

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