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Lipid–ASO therapeutics exhibit differential tissue targeted delivery upon systemic or local CNS administration

S. Roudi H. Y. Estupiñán O. Saher Cristiana Barradas Emma Inganäs Hoang-Ngoan Le N. Frengen Radosław Grochowski Svetlana Pavlova Robert Månsson Welinder Joel Z. Nordin Annabelle Biscans Pär Matsson R. Zain R. Sandberg Michael Hagemann-Jensen S. el Andaloussi

Sep 2026 · bioRxiv · 0 citations · 59 references

Biology

TL;DR

It is demonstrated that lipid identity critically determines ASO efficacy, productive cellular uptake, and regional CNS engagement, emphasizing the need for context-specific lipid design in ASO therapeutic development.

DOI Semantic Scholar

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