Advanced siRNA Designs Further Improve In Vivo Performance of GalNAc-siRNA Conjugates

Significant progress has been made in the advancement of RNAi therapeutics by combining a synthetic triantennary N-acetylgalactosamine ligand targeting the asialoglycoprotein receptor with chemically modified small interfering RNA (siRNA) designs, including the recently described Enhanced Stabilizat...

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Veröffentlicht in:Mol Ther
Hauptverfasser: Foster, Donald J., Brown, Christopher R., Shaikh, Sarfraz, Trapp, Casey, Schlegel, Mark K., Qian, Kun, Sehgal, Alfica, Rajeev, Kallanthottathil G., Jadhav, Vasant, Manoharan, Muthiah, Kuchimanchi, Satya, Maier, Martin A., Milstein, Stuart
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Society of Gene & Cell Therapy 2018
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Original Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5910670/
https://ncbi.nlm.nih.gov/pubmed/29456020
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.12.021
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