Correlating in vitro splice switching activity with systemic in vivo delivery using novel ZEN-modified oligonucleotides

Molecular Therapy Nucleic Acids

Volumn 3, Issue , 2014, Pages e212-

  Hammond, Suzan M ^a   McClorey, Graham ^a   Nordin, Joel Z ^b   Godfrey, Caroline ^a   Stenler, Sofia ^b   Lennox, Kim A ^c   Smith, C I Edvard ^b   Jacobi, Ashley M ^c   Varela, Miguel A ^a   Lee, Yi ^a   Behlke, Mark A ^c   Wood, Matthew J A ^a   Andaloussi, Samir E L ^a,b  

^a UNIVERSITY OF OXFORD   (United Kingdom)

^b KAROLINSKA UNIVERSITY HOSPITAL   (Sweden)

^c INTEGRATED DNA TECHNOLOGIES   (United States)

Author keywords

Antisense oligonucleotides; Duchenne muscular dystrophy; Splice switching; Zen modification

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; DYSTROPHIN; PHOSPHOROTHIOIC ACID;

ALTERNATIVE RNA SPLICING; ARTICLE; BINDING AFFINITY; CHEMICAL MODIFICATION; EXON SKIPPING; GENE INSERTION; IN VITRO STUDY; IN VIVO STUDY; MOLECULAR THERAPY; NONHUMAN; PRIORITY JOURNAL;

EID: 84925273134     PISSN: None     EISSN: 21622531     Source Type: Journal    
DOI: 10.1038/mtna.2014.63     Document Type: Article

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