Somatic therapy of a mouse SMA model with a U7 snRNA gene correcting SMN2 splicing

Molecular Therapy

Volumn 24, Issue 10, 2016, Pages 1797-1805

  Odermatt, Philipp ^a   Trüb, Judith ^a   Furrer, Lavinia ^a   Fricker, Roger ^a   Marti, Andreas ^a   Schümperli, Daniel ^a  

^a UNIVERSITY OF BERN   (Switzerland)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; SMALL NUCLEAR RNA; SURVIVAL MOTOR NEURON PROTEIN 2; U7 SMALL NUCLEAR RNA; UNCLASSIFIED DRUG; SMN2 PROTEIN, MOUSE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CONTROLLED STUDY; DISEASE SEVERITY; GENE CASSETTE; GENE EXPRESSION; GENE FREQUENCY; GENETIC ASSOCIATION; MOTONEURON; MOUSE; NEWBORN; NONHUMAN; PHENOTYPE; PROCESS OPTIMIZATION; RNA SPLICING; SMN2 GENE; SOMATIC GENE THERAPY; SPINAL MUSCULAR ATROPHY; VIRAL GENE THERAPY; VIRUS PARTICLE; ADENOVIRIDAE; ADMINISTRATION AND DOSAGE; ANIMAL; CARDIAC MUSCLE; DISEASE MODEL; GENE THERAPY; GENE VECTOR; GENETICS; LIVER; METABOLISM; PROCEDURES; TRANSGENIC MOUSE;

ADENOVIRIDAE; ANIMALS; DISEASE MODELS, ANIMAL; GENETIC THERAPY; GENETIC VECTORS; LIVER; MICE; MICE, TRANSGENIC; MUSCULAR ATROPHY, SPINAL; MYOCARDIUM; RNA SPLICING; RNA, SMALL NUCLEAR; SURVIVAL OF MOTOR NEURON 2 PROTEIN;

EID: 84984783367     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2016.152     Document Type: Article

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