Autor: Lyon, Alexander R. - Prolib Integro

Skocz do pozycji: 2.

Tytuł:: Safer and efficient base editing and prime editing via ribonucleoproteins delivered through optimized lipid-nanoparticle formulations
Autorzy:: Palczewski, Krzysztof
Chen, Paul Z.
Kiser, Philip D.
Risalit,i Eleonora
Rodrigues Menezes, Carolline
Hołubowicz, Rafał
Smidak, Roman
Griffo Alessandra
Caprettini Valeria
Felgner, Philip L.
Dong, Zhiqian
Liu, David R.
Almeida Filipe
Lyon, David C.
Cheifet Barbara
Salom, David
Felgner, Jiin
Choi, Elliot H.
Palczewska, Grazyna
Bassetto, Marco
Du, Samuel W.
Haskell Jennifer
Gao, Fangyuan
Medani, Omar
Yan, Alexander L.
Hołubowicz, Maria W.
Newby, Gregory A.
Foik, Andrzej T.
Workman, J. Noah
Współwytwórcy:: Caprettini Valeria
Almeida Filipe
Cheifet Barbara
Griffo Alessandra
Haskell Jennifer
Wydawca:: Nature Portfolio
Cytata wydawnicza:: Hołubowicz, R., Du, S.W., Felgner, J. et al. Safer and efficient base editing and prime editing via ribonucleoproteins delivered through optimized lipid-nanoparticle formulations. Nat. Biomed. Eng 9, 57–78 (2025). https://doi.org/10.1038/s41551-024-01296-2
Opis:: Delivering ribonucleoproteins (RNPs) for in vivo genome editing is safer than using viruses encoding for Cas9 and its respective guide RNA. However, transient RNP activity does not typically lead to optimal editing outcomes. Here we show that the efficiency of delivering RNPs can be enhanced by cell-penetrating peptides (covalently fused to the protein or as excipients) and that lipid nanoparticles (LNPs) encapsulating RNPs can be optimized for enhanced RNP stability, delivery efficiency and editing potency. Specifically, after screening for suitable ionizable cationic lipids and by optimizing the concentration of the synthetic lipid DMG-PEG 2000, we show that the encapsulation, via microfluidic mixing, of adenine base editor and prime editor RNPs within LNPs using the ionizable lipid SM102 can result in in vivo editing-efficiency enhancements larger than 300-fold (with respect to the delivery of the naked RNP) without detectable off-target edits. We believe that chemically defined LNP formulations optimized for RNP-encapsulation stability and delivery efficiency will lead to safer genome editing.
Dostawca treści:: Repozytorium Centrum Otwartej Nauki

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