Selection of GalNAc-conjugated siRNAs with limited off-target-driven rat hepatotoxicity

Small interfering RNAs (siRNAs) conjugated to a trivalent N-acetylgalactosamine (GalNAc) ligand are being evaluated in investigational clinical studies for a variety of indications. The typical development candidate selection process includes evaluation of the most active compounds for toxicity in r...

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Publicat a:Nat Commun
Autors principals: Janas, Maja M., Schlegel, Mark K., Harbison, Carole E., Yilmaz, Vedat O., Jiang, Yongfeng, Parmar, Rubina, Zlatev, Ivan, Castoreno, Adam, Xu, Huilei, Shulga-Morskaya, Svetlana, Rajeev, Kallanthottathil G., Manoharan, Muthiah, Keirstead, Natalie D., Maier, Martin A., Jadhav, Vasant
Format: Artigo
Idioma:Inglês
Publicat: Nature Publishing Group UK 2018
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC5818625/
https://ncbi.nlm.nih.gov/pubmed/29459660
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41467-018-02989-4
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