Expanding Conjugate Space of RNAi Therapeutics: Ligand at the 3′ End of the Antisense Strand Achieves Uncompromised In Vivo Potency and Efficacy and Reveals Interactions with the Argonaute-2 PAZ Domain

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DOI:10.1021/acs.jmedchem.4c02250
作者：Rajat S. Das, Dhrubajyoti Datta, Christopher R. Brown, Jason A. Gilbert, Amy Chan, Jennifer Willoughby, Swati Gupta, MaryBeth Kim, Rohan Degaonkar, Tim Racie, Li Lei, Mark K. Schlegel, Adam Castoreno,
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American Chemical Society (ACS)

Abstract：The conjugation of the sense strands of small interfering RNA (siRNA) to tri-N-acetylgalactosamine (GalNAc), the ligand for a hepatocyte-specific receptor, enables the delivery of multiple c

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