Delivery of GalNAc-Conjugated Splice-Switching ASOs to Non-hepatic Cells through Ectopic Expression of Asialoglycoprotein Receptor

Splice-switching antisense oligonucleotides (ASOs) are promising therapeutic tools to target various genetic diseases, including cancer. However, in vivo delivery of ASOs to orthotopic tumors in cancer mouse models or to certain target tissues remains challenging. A viable solution already in use is...

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Detalles Bibliográficos
Publicado en:Mol Ther Nucleic Acids
Main Authors: Scharner, Juergen, Qi, Sabrina, Rigo, Frank, Bennett, C. Frank, Krainer, Adrian R.
Formato: Artigo
Idioma:Inglês
Publicado: American Society of Gene & Cell Therapy 2019
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Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC6453860/
https://ncbi.nlm.nih.gov/pubmed/30965276
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2019.02.024
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