Correlating In Vitro Splice Switching Activity With Systemic In Vivo Delivery Using Novel ZEN-modified Oligonucleotides

• Main Authors: Suzan M Hammond (Author), Graham McClorey (Author), Joel Z Nordin (Author), Caroline Godfrey (Author), Sofia Stenler (Author), Kim A Lennox (Author), CI Edvard Smith (Author), Ashley M Jacobi (Author), Miguel A Varela (Author), Yi Lee (Author), Mark A Behlke (Author), Matthew J A Wood (Author), Samir E L Andaloussi (Author)
• Format: Book
• Published: Elsevier, 2014-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Splice switching oligonucleotides (SSOs) induce alternative splicing of pre-mRNA and typically employ chemical modifications to increase nuclease resistance and binding affinity to target pre-mRNA. Here we describe a new SSO non-base modifier (a naphthyl-azo group, "ZEN™") to direct exon e...