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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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  Data: Single alkyl phosphonate modification of the siRNA backbone in the seed region enhances specificity and therapeutic profile.
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  Data: <searchLink fieldCode="AU" term="%22Nikan+M%22">Nikan M</searchLink>; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States.<br /><searchLink fieldCode="AU" term="%22Li+Q%22">Li Q</searchLink>; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States.<br /><searchLink fieldCode="AU" term="%22Tanowitz+M%22">Tanowitz M</searchLink>; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States.<br /><searchLink fieldCode="AU" term="%22Li+H%22">Li H</searchLink>; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States.<br /><searchLink fieldCode="AU" term="%22Damle+S%22">Damle S</searchLink>; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States.<br /><searchLink fieldCode="AU" term="%22Annoual+M%22">Annoual M</searchLink>; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States.<br /><searchLink fieldCode="AU" term="%22Galindo-Murillo+R%22">Galindo-Murillo R</searchLink>; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States.<br /><searchLink fieldCode="AU" term="%22Low+A%22">Low A</searchLink>; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States.<br /><searchLink fieldCode="AU" term="%22Klein+S%22">Klein S</searchLink>; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States.<br /><searchLink fieldCode="AU" term="%22Quirk+C%22">Quirk C</searchLink>; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States.<br /><searchLink fieldCode="AU" term="%22Vasquez+G%22">Vasquez G</searchLink>; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States.<br /><searchLink fieldCode="AU" term="%22Wan+WB%22">Wan WB</searchLink>; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States.<br /><searchLink fieldCode="AU" term="%22Watt+AT%22">Watt AT</searchLink>; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States.<br /><searchLink fieldCode="AU" term="%22Migawa+MT%22">Migawa MT</searchLink>; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States.<br /><searchLink fieldCode="AU" term="%22Swayze+EE%22">Swayze EE</searchLink>; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States.<br /><searchLink fieldCode="AU" term="%22Prakash+TP%22">Prakash TP</searchLink>; Ionis Pharmaceuticals Inc., 2855 Gazelle Ct, Carlsbad, CA 92010, United States.
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              Text: 2025 Jul 19
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