Shorter Is Better: The α-(<scp>l</scp>)-Threofuranosyl Nucleic Acid Modification Improves Stability, Potency, Safety, and Ago2 Binding and Mitigates Off-Target Effects of Small Interfering RNAs-Reference-Cited by-同舟云学术

Shorter Is Better: The α-(l)-Threofuranosyl Nucleic Acid Modification Improves Stability, Potency, Safety, and Ago2 Binding and Mitigates Off-Target Effects of Small Interfering RNAs

Published:2023-08-28 Issue:36 Volume:145 Page:19691-19706
ISSN:0002-7863
Container-title:Journal of the American Chemical Society
language:en
Short-container-title:J. Am. Chem. Soc.

Author:

Matsuda Shigeo¹,Bala Saikat²^ORCID,Liao Jen-Yu²,Datta Dhrubajyoti¹,Mikami Atsushi¹,Woods Lauren¹,Harp Joel M.³,Gilbert Jason A.¹,Bisbe Anna¹,Manoharan Rajar M.¹,Kim MaryBeth¹,Theile Christopher S.¹,Guenther Dale C.¹,Jiang Yongfeng¹,Agarwal Saket¹^ORCID,Maganti Rajanikanth¹,Schlegel Mark K.¹^ORCID,Zlatev Ivan¹,Charisse Klaus¹,Rajeev Kallanthottathil G.¹,Castoreno Adam¹,Maier Martin¹,Janas Maja M.¹,Egli Martin³^ORCID,Chaput John C.²^ORCID,Manoharan Muthiah¹^ORCID

Affiliation:

1. Alnylam Pharmaceuticals, 675 West Kendall Street, Cambridge, Massachusetts 02142, United States

2. Department of Pharmaceutical Sciences, University of California, Irvine, California 92697-3958, United States

3. Department of Biochemistry, School of Medicine, Vanderbilt University, Nashville, Tennessee 37232-0146, United States

Publisher

American Chemical Society (ACS)

Subject

Colloid and Surface Chemistry,Biochemistry,General Chemistry,Catalysis

Link

https://pubs.acs.org/doi/pdf/10.1021/jacs.3c04744

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1. The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs

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5. Inclisiran for the Treatment of Heterozygous Familial Hypercholesterolemia

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