Postsymptomatic restoration of SMN rescues the disease phenotype in a mouse model of severe spinal muscular atrophy

Journal of Clinical Investigation

Volumn 121, Issue 8, 2011, Pages 3029-3041

  Lutz, Cathleen M ^a   Kariya, Shingo ^b   Patruni, Sunita ^b   Osborne, Melissa A ^a   Liu, Don ^a   Henderson, Christopher E ^b   Li, Darrick K ^b   Pellizzoni, Livio ^b   Rojas, José ^c   Valenzuela, David M ^c   Murphy, Andrew J ^c   Winberg, Margaret L ^d   Monani, Umrao R ^b,e  

^a JACKSON LABORATORY   (United States)

^b NewYork Presbyterian Hospital   (United States)

^c REGENERON PHARMACEUTICALS INC   (United States)

^d Spinal Muscular Atrophy Foundation   (United States)

^e P and S   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

SURVIVAL MOTOR NEURON PROTEIN; SURVIVAL MOTOR NEURON PROTEIN 1; SURVIVAL MOTOR NEURON PROTEIN 2;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CONTROLLED STUDY; DISEASE COURSE; DISEASE SEVERITY; MOTONEURON; MOUSE; NEUROMUSCULAR SYNAPSE; NONHUMAN; PHENOTYPE; PRIORITY JOURNAL; PROTEIN EXPRESSION; SPINAL MUSCULAR ATROPHY;

ALLELES; ANIMALS; CLONING, MOLECULAR; CROSSES, GENETIC; DISEASE MODELS, ANIMAL; EXTREMITIES; HUMANS; MICE; MOTOR NEURONS; MUSCULAR ATROPHY, SPINAL; PHENOTYPE; REFLEX; SURVIVAL OF MOTOR NEURON 1 PROTEIN; SYNAPSES; TREATMENT OUTCOME;

EID: 79960987691     PISSN: 00219738     EISSN: 15588238     Source Type: Journal    
DOI: 10.1172/JCI57291     Document Type: Article

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