Design and application of bispecific splice-switching oligonucleotides

Nucleic Acid Therapeutics

Volumn 24, Issue 1, 2014, Pages 13-24

  Bestas, Burcu ^a   McClorey, Graham ^b   Tedebark, Ulf ^a,c   Moreno, Pedro M D ^a,e   Roberts, Thomas C ^b,d   Hammond, Suzan M ^b   Smith, C I Edvard ^a   Wood, Matthew J A ^b   Andaloussi, Samir El ^a,b  

^a KAROLINSKA INSTITUTE   (Sweden)

^b UNIVERSITY OF OXFORD   (United Kingdom)

^c GE Healthcare Bio Sciences AB   (Sweden)

^d SCRIPPS RESEARCH INSTITUTE   (United States)

^e UNIVERSITY OF PORTO   (Portugal)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; DISULFIDE; DYSTROPHIN; HYDROCARBON; MESSENGER RNA PRECURSOR; MYOSTATIN; SPLICE SWITCHING OLIGONUCLEOTIDE; UNCLASSIFIED DRUG;

ANIMAL CELL; ANTISENSE THERAPY; ARTICLE; CELL VIABILITY; CONTROLLED STUDY; DRUG CONJUGATION; DRUG DESIGN; DRUG EFFICACY; DRUG SCREENING; DUCHENNE MUSCULAR DYSTROPHY; EXON SKIPPING; FLUORESCENCE ACTIVATED CELL SORTING; GENE DISRUPTION; GENE EXPRESSION; GENE SWITCHING; IN VITRO GENE TRANSFER; MOUSE; MUSCLE HYPERTROPHY; MYOTUBE; NONHUMAN; NUCLEIC ACID SYNTHESIS; OPEN READING FRAME; PRIORITY JOURNAL; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; RNA EXTRACTION; RNA SPLICING; SOLID PHASE SYNTHESIS;

ANIMALS; BASE SEQUENCE; CELL LINE; DYSTROPHIN; EXONS; HUMANS; MALE; MICE; MICE, INBRED C57BL; MICE, INBRED MDX; MUSCULAR DYSTROPHY, ANIMAL; MUSCULAR DYSTROPHY, DUCHENNE; MUTATION; MYOSTATIN; OLIGONUCLEOTIDES, ANTISENSE; RNA SPLICING; TARGETED GENE REPAIR;

EID: 84893600393     PISSN: 21593337     EISSN: None     Source Type: Journal    
DOI: 10.1089/nat.2013.0462     Document Type: Article

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