Strategies for In Vivo Screening and Mitigation of Hepatotoxicity Associated with Antisense Drugs

Antisense oligonucleotide (ASO) gapmers downregulate gene expression by inducing enzyme-dependent degradation of targeted RNA and represent a promising therapeutic platform for addressing previously undruggable genes. Unfortunately, their therapeutic application, particularly that of the more potent...

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Detalles Bibliográficos
Autores principales: Kamola, Piotr J., Maratou, Klio, Wilson, Paul A., Rush, Kay, Mullaney, Tanya, McKevitt, Tom, Evans, Paula, Ridings, Jim, Chowdhury, Probash, Roulois, Aude, Fairchild, Ann, McCawley, Sean, Cartwright, Karen, Gooderham, Nigel J., Gant, Timothy W., Moores, Kitty, Hughes, Stephen A., Edbrooke, Mark R., Clark, Kenneth, Parry, Joel D.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2017
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5537172/
https://www.ncbi.nlm.nih.gov/pubmed/28918038
http://dx.doi.org/10.1016/j.omtn.2017.07.003

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5537172/
https://www.ncbi.nlm.nih.gov/pubmed/28918038
http://dx.doi.org/10.1016/j.omtn.2017.07.003

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