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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.

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Title: 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.
Authors: Matsuda S; Alnylam Pharmaceuticals, 675 West Kendall Street, Cambridge, Massachusetts 02142, United States., Bala S; Department of Pharmaceutical Sciences, University of California, Irvine, California 92697-3958, United States., Liao JY; Department of Pharmaceutical Sciences, University of California, Irvine, California 92697-3958, United States., Datta D; Alnylam Pharmaceuticals, 675 West Kendall Street, Cambridge, Massachusetts 02142, United States., Mikami A; Alnylam Pharmaceuticals, 675 West Kendall Street, Cambridge, Massachusetts 02142, United States., Woods L; Alnylam Pharmaceuticals, 675 West Kendall Street, Cambridge, Massachusetts 02142, United States., Harp JM; Department of Biochemistry, School of Medicine, Vanderbilt University, Nashville, Tennessee 37232-0146, United States., Gilbert JA; Alnylam Pharmaceuticals, 675 West Kendall Street, Cambridge, Massachusetts 02142, United States., Bisbe A; Alnylam Pharmaceuticals, 675 West Kendall Street, Cambridge, Massachusetts 02142, United States., Manoharan RM; Alnylam Pharmaceuticals, 675 West Kendall Street, Cambridge, Massachusetts 02142, United States., Kim M; Alnylam Pharmaceuticals, 675 West Kendall Street, Cambridge, Massachusetts 02142, United States., Theile CS; Alnylam Pharmaceuticals, 675 West Kendall Street, Cambridge, Massachusetts 02142, United States., Guenther DC; Alnylam Pharmaceuticals, 675 West Kendall Street, Cambridge, Massachusetts 02142, United States., Jiang Y; Alnylam Pharmaceuticals, 675 West Kendall Street, Cambridge, Massachusetts 02142, United States., Agarwal S; Alnylam Pharmaceuticals, 675 West Kendall Street, Cambridge, Massachusetts 02142, United States., Maganti R; Alnylam Pharmaceuticals, 675 West Kendall Street, Cambridge, Massachusetts 02142, United States., Schlegel MK; Alnylam Pharmaceuticals, 675 West Kendall Street, Cambridge, Massachusetts 02142, United States., Zlatev I; Alnylam Pharmaceuticals, 675 West Kendall Street, Cambridge, Massachusetts 02142, United States., Charisse K; Alnylam Pharmaceuticals, 675 West Kendall Street, Cambridge, Massachusetts 02142, United States., Rajeev KG; Alnylam Pharmaceuticals, 675 West Kendall Street, Cambridge, Massachusetts 02142, United States., Castoreno A; Alnylam Pharmaceuticals, 675 West Kendall Street, Cambridge, Massachusetts 02142, United States., Maier M; Alnylam Pharmaceuticals, 675 West Kendall Street, Cambridge, Massachusetts 02142, United States., Janas MM; Alnylam Pharmaceuticals, 675 West Kendall Street, Cambridge, Massachusetts 02142, United States., Egli M; Department of Biochemistry, School of Medicine, Vanderbilt University, Nashville, Tennessee 37232-0146, United States., Chaput JC; Department of Pharmaceutical Sciences, University of California, Irvine, California 92697-3958, United States., Manoharan M; Alnylam Pharmaceuticals, 675 West Kendall Street, Cambridge, Massachusetts 02142, United States.
Source: Journal of the American Chemical Society [J Am Chem Soc] 2023 Sep 13; Vol. 145 (36), pp. 19691-19706. Date of Electronic Publication: 2023 Aug 28.
Publication Type: Journal Article
Journal Info: Publisher: American Chemical Society Country of Publication: United States NLM ID: 7503056 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1520-5126 (Electronic) Linking ISSN: 00027863 NLM ISO Abbreviation: J Am Chem Soc Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1520-5126
DOI:10.1021/jacs.3c04744