Antibody-oligonucleotide conjugates (AOCs) offer a promising solution to delivery challenges of therapeutic oligonucleotides. However, the relationship between their complex chemical architectures and biological activity remains poorly understood, limiting the development of important structure-function relationships....
O. Hayes, J. Rädler, E. Filipiak et al.· bioRxiv· 0 citations
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.
S. Roudi, H. Y. Estupiñán, O. Saher et al.· bioRxiv· 0 citations
It is shown that an optimized single-guide RNA scaffold architecture improves RNP stability, and when combined with additional EV engineering leads to a three-hundred-fold increase in potency, enabling efficient base editing or knockout in primary cells, human brain organoids and in vivo, including the mouse brain.
Xiuming Liang, Houze Zhou, Z. Nizamudeen et al.· bioRxiv· 0 citations
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