Gene therapy rescues disease phenotype in a spinal muscular atrophy with respiratory distress type 1 (SMARD1) mouse model

Science Advances

Volumn 1, Issue 2, 2015, Pages

  Nizzardo, Monica ^a   Simone, Chiara ^a   Rizzo, Federica ^a   Salani, Sabrina ^a   Dametti, Sara ^a   Rinchetti, Paola ^a   Del Bo, Roberto ^a   Foust, Kevin ^b   Kaspar, Brian K ^b,c   Bresolin, Nereo ^a   Comi, Giacomo P ^a   Corti, Stefania ^a  

^a UNIVERSITY OF MILAN   (Italy)

^b OHIO STATE UNIVERSITY   (United States)

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

Author keywords

[No Author keywords available]

Indexed keywords

BRAIN; DISEASE CONTROL; GENE ENCODING; GENES; MAMMALS; MEDICAL APPLICATIONS; NEUROMUSCULAR REHABILITATION; NEURONS; PHYSIOLOGICAL MODELS; STEM CELLS; VIRUSES;

ADENO-ASSOCIATED VIRUS; CENTRAL NERVOUS SYSTEMS; DISEASE PHENOTYPES; INDUCED PLURIPOTENT STEM CELLS; LONG-TERM CULTURE; MOTOR NEURON DISEASE; SPINAL MUSCULAR ATROPHY; THERAPEUTIC DELIVERY;

GENE THERAPY;

EID: 84969852911     PISSN: None     EISSN: 23752548     Source Type: Journal    
DOI: 10.1126/sciadv.1500078     Document Type: Article

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