Optimization of morpholino antisense oligonucleotides targeting the intronic repressor element1 in spinal muscular atrophy

Molecular Therapy

Volumn 24, Issue 9, 2016, Pages 1592-1601

  Osman, Erkan Y ^a   Washington, Charles W ^a   Kaifer, Kevin A ^a,b   Mazzasette, Chiara ^c   Patitucci, Teresa N ^d   Florea, Kyra M ^a,b   Simon, Madeline E ^a,b   Ko, Chien Ping ^c   Ebert, Allison D ^d   Lorson, Christian L ^a,b  

^a UNIVERSITY OF MISSOURI   (United States)

^b University of Missouri   (United States)

^c UNIVERSITY OF SOUTHERN CALIFORNIA   (United States)

^d MEDICAL COLLEGE OF WISCONSIN   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGODEOXYNUCLEOTIDE; SURVIVAL MOTOR NEURON PROTEIN 2; MESSENGER RNA; MORPHOLINO OLIGONUCLEOTIDE; SURVIVAL MOTOR NEURON PROTEIN 1;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DISEASE SEVERITY; DNA SPLICING; DOSE RESPONSE; DRUG EFFICACY; GENE STRUCTURE; HUMAN; HUMAN CELL; IN VIVO STUDY; INTRON; INTRONIC REPRESSOR ELEMENT1; LIFESPAN; MOUSE; NONHUMAN; NUCLEOTIDE SEQUENCE; SINGLE DRUG DOSE; SMN2 GENE; SPINAL MUSCULAR ATROPHY; ANIMAL; CYTOLOGY; DISEASE MODEL; DNA RESPONSIVE ELEMENT; GENE EXPRESSION REGULATION; GENE TARGETING; GENETIC TRANSCRIPTION; GENETICS; INDUCED PLURIPOTENT STEM CELL; KNOCKOUT MOUSE; METABOLISM; MORTALITY; MUTATION; PROGNOSIS;

ANIMALS; DISEASE MODELS, ANIMAL; GENE EXPRESSION REGULATION; GENE TARGETING; HUMANS; INDUCED PLURIPOTENT STEM CELLS; INTRONS; MICE; MICE, KNOCKOUT; MORPHOLINOS; MUSCULAR ATROPHY, SPINAL; MUTATION; PROGNOSIS; RESPONSE ELEMENTS; RNA, MESSENGER; SURVIVAL OF MOTOR NEURON 1 PROTEIN; TRANSCRIPTION, GENETIC;

EID: 84981277292     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2016.145     Document Type: Article

References (46)

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