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

Reference59 articles.

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

2. Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria

3. Preclinical Development of a Subcutaneous ALAS1 RNAi Therapeutic for Treatment of Hepatic Porphyrias Using Circulating RNA Quantification

4. An Investigational RNAi Therapeutic Targeting Glycolate Oxidase Reduces Oxalate Production in Models of Primary Hyperoxaluria

5. Inclisiran for the Treatment of Heterozygous Familial Hypercholesterolemia

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