TSUNAMI: An antisense method to phenocopy splicing-associated diseases in animals

Genes and Development

Volumn 26, Issue 16, 2012, Pages 1874-1884

  Sahashi, Kentaro ^a   Hua, Yimin ^a   Ling, Karen K Y ^b   Hung, Gene ^c   Rigo, Frank ^c   Horev, Guy ^a   Katsuno, Masahisa ^d   Sobue, Gen ^d   Ko, Chien Ping ^b   Frank Bennett, C ^c   Krainer, Adrian R ^a  

^a Howard Hughes Medical Institute Cold Spring Harbor Laboratory Cold Spring Harbor   (United States)

^b UNIVERSITY OF SOUTHERN CALIFORNIA   (United States)

^c ISIS PHARMACEUTICALS   (United States)

^d NAGOYA UNIVERSITY GRADUATE SCHOOL OF MEDICINE   (Japan)

Author keywords

Antisense oligonucleotide; Mouse model; Phenocopy; SMN2; Spinal muscular atrophy; Splicing modulation

Indexed keywords

2' O (2 METHOXYETHYL); ANTISENSE OLIGONUCLEOTIDE; ANTISENSE OLIGONUCLEOTIDE[10-27]; ANTISENSE OLIGONUCLEOTIDE[20-37]; ANTISENSE OLIGONUCLEOTIDE[23-42]; MESSENGER RNA; SURVIVAL MOTOR NEURON PROTEIN 2; UNCLASSIFIED DRUG;

ALPHA NERVE FIBER; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; DISEASE COURSE; EXON SKIPPING; GROWTH DISORDER; LIFESPAN; MOTOR DYSFUNCTION; MOUSE; NEUROMUSCULAR SYNAPSE; NEWBORN; NONHUMAN; PATHOGENESIS; PHENOTYPE; PRIORITY JOURNAL; RNA SPLICING; SPINAL MUSCULAR ATROPHY; TRANSGENIC MOUSE; TSUNAMI METHOD;

ANIMALS; GENE THERAPY; GENETIC TECHNIQUES; MICE; MICE, TRANSGENIC; MUSCULAR ATROPHY, SPINAL; OLIGONUCLEOTIDES, ANTISENSE; RNA SPLICING; SURVIVAL OF MOTOR NEURON 1 PROTEIN; SURVIVAL OF MOTOR NEURON 2 PROTEIN;

EID: 84865079103     PISSN: 08909369     EISSN: 15495477     Source Type: Journal    
DOI: 10.1101/gad.197418.112     Document Type: Article

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