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Single alkyl phosphonate modification of the siRNA backbone in the seed region enhances specificity and therapeutic profile.

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Title: Single alkyl phosphonate modification of the siRNA backbone in the seed region enhances specificity and therapeutic profile.
Authors: Nikan M; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States., Li Q; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States., Tanowitz M; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States., Li H; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States., Damle S; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States., Annoual M; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States., Galindo-Murillo R; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States., Low A; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States., Klein S; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States., Quirk C; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States., Vasquez G; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States., Wan WB; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States., Watt AT; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States., Migawa MT; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States., Swayze EE; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States., Prakash TP; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States.
Source: Nucleic acids research [Nucleic Acids Res] 2025 Jul 19; Vol. 53 (14).
Publication Type: Journal Article
Journal Info: Publisher: Oxford University Press Country of Publication: England NLM ID: 0411011 Publication Model: Print Cited Medium: Internet ISSN: 1362-4962 (Electronic) Linking ISSN: 03051048 NLM ISO Abbreviation: Nucleic Acids Res Subsets: MEDLINE
Database: MEDLINE Ultimate
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Description
ISSN:1362-4962
DOI:10.1093/nar/gkaf692