Spinal muscular atrophy phenotype is ameliorated in human motor neurons by SMN increase via different novel RNA therapeutic approaches

Scientific Reports

Volumn 5, Issue , 2015, Pages

  Nizzardo, Monica ^a   Simone, Chiara ^a   Dametti, Sara ^a   Salani, Sabrina ^a   Ulzi, Gianna ^a   Pagliarani, Serena ^a   Rizzo, Federica ^a   Frattini, Emanuele ^a   Pagani, Franco ^b   Bresolin, Nereo ^a   Comi, Giacomo ^a   Corti, Stefania ^a  

^a UNIVERSITY OF MILAN   (Italy)

^b INTERNATIONAL CENTRE FOR GENETIC ENGINEERING AND BIOTECHNOLOGY   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

MORPHOLINO OLIGONUCLEOTIDE; SMALL NUCLEAR RNA; SMN2 PROTEIN, HUMAN; SURVIVAL MOTOR NEURON PROTEIN 2; TRANSCRIPTION FACTOR; U1 SMALL NUCLEAR RNA;

CELL CULTURE; CELL SURVIVAL; FEMALE; GENE EXPRESSION REGULATION; GENE SILENCING; GENE THERAPY; GENETICS; HUMAN; INDUCED PLURIPOTENT STEM CELL; MALE; METABOLISM; MOTONEURON; MUSCULAR ATROPHY, SPINAL; NUCLEOTIDE SEQUENCE; PHENOTYPE; PHYSIOLOGY; PROMOTER REGION;

BASE SEQUENCE; CELL SURVIVAL; CELLS, CULTURED; FEMALE; GENE EXPRESSION REGULATION; GENE KNOCKDOWN TECHNIQUES; GENETIC THERAPY; HUMANS; INDUCED PLURIPOTENT STEM CELLS; MALE; MORPHOLINOS; MOTOR NEURONS; MUSCULAR ATROPHY, SPINAL; PHENOTYPE; PROMOTER REGIONS, GENETIC; RNA, SMALL NUCLEAR; SURVIVAL OF MOTOR NEURON 2 PROTEIN; TRANSCRIPTION FACTORS;

EID: 84934783032     PISSN: None     EISSN: 20452322     Source Type: Journal    
DOI: 10.1038/srep11746     Document Type: Article

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