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

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 exclusion i...

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Detalles Bibliográficos
Autores principales: Hammond, Suzan M, McClorey, Graham, Nordin, Joel Z, Godfrey, Caroline, Stenler, Sofia, Lennox, Kim A, Smith, CI Edvard, Jacobi, Ashley M, Varela, Miguel A, Lee, Yi, Behlke, Mark A, Wood, Matthew J A, Andaloussi, Samir E L
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2014
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4459549/
https://www.ncbi.nlm.nih.gov/pubmed/25423116
http://dx.doi.org/10.1038/mtna.2014.63

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4459549/
https://www.ncbi.nlm.nih.gov/pubmed/25423116
http://dx.doi.org/10.1038/mtna.2014.63

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