Bifunctional RNAs targeting the intronic splicing silencer N1 increase SMN levels and reduce disease severity in an animal model of spinal muscular atrophy

Molecular Therapy

Volumn 20, Issue 1, 2012, Pages 119-126

  Osman, Erkan Y ^a,b   Yen, Pei Fen ^b   Lorson, Christian L ^a,b  

^a University of Missouri   (United States)

^b UNIVERSITY OF MISSOURI   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

2' O METHYL RNA; COMPLEMENTARY DNA; COMPLEMENTARY RNA; RNA; SURVIVAL MOTOR NEURON PROTEIN 1; SURVIVAL MOTOR NEURON PROTEIN 2; UNCLASSIFIED DRUG;

ALTERNATIVE RNA SPLICING; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTISENSE THERAPY; ARTICLE; CONTROLLED STUDY; DISEASE SEVERITY; DOSE RESPONSE; DRUG DOSE INCREASE; DRUG EFFECT; DRUG MECHANISM; EXON; GENE; GENE EXPRESSION REGULATION; GENE TARGETING; HUMAN; HUMAN CELL; INTRON; INTRONIC SPLICING SILENCER N1; MOUSE; NEWBORN; NONHUMAN; OUTCOME ASSESSMENT; PHENOTYPE; PROTEIN EXPRESSION; PROTEIN LOCALIZATION; SILENCER ELEMENT; SPINAL MUSCULAR ATROPHY; SURVIVAL MOTOR NEURON 1 GENE; SURVIVAL MOTOR NEURON 2 GENE; SURVIVAL TIME; UPREGULATION; WEIGHT GAIN;

ANIMALS; BASE SEQUENCE; DISEASE MODELS, ANIMAL; GENE ORDER; GENE TARGETING; GENE THERAPY; INTRONS; MICE; MICE, KNOCKOUT; MOTOR ACTIVITY; MUSCULAR ATROPHY, SPINAL; OLIGORIBONUCLEOTIDES, ANTISENSE; REGULATORY SEQUENCES, RIBONUCLEIC ACID; RNA SPLICING; SMN COMPLEX PROTEINS; WEIGHT GAIN;

EID: 84856970439     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2011.232     Document Type: Article

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