9d ago
Lipid-ASO therapeutics exhibit differential tissue targeted delivery upon systemic or local CNS administration
Roudi · S. · Estupinan · H. Y. · Saher · O. · Barradas · C. · +26 more
Abstract
Antisense oligonucleotides (ASOs) are a powerful therapeutic modality, but their full potential is hindered by pharmacokinetic properties that affect tissue and cellular delivery. Lipid conjugation is increasingly used to modulate ASO's biodistribution and promote extrahepatic activity, yet lipid dependent effects on in vivo functional delivery, particularly in the central nervous system (CNS), remain less explored. Here, we performed a side by side in vivo comparison of cholesterol, palmitic ac
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