Safer and efficient base editing and prime editing via ribonucleoproteins delivered through optimized lipid-nanoparticle formulations

Hołubowicz, Rafał; W. Du, Samuel; Felgner, Jiin; Šmidák, Roman; H. Choi, Elliot; Palczewska, Grażyna; Rodrigues Menezes, Carolline; Dong, Zhiqian; Gao, Fangyuan; Medani, Omar; L. Yan, Alexander; W. Hołubowicz, Maria; Chen, Paul; Bassetto, Marco; Risaliti, Eleonora; Salom, David; Noah Workman, J.; D. Kiser, Philip; T. Foik, Andrzej; C. Lyon, David; A. Newby, Gregory; R. Liu, David; L. Felgner, Philip; Palczewski, Krzysztof

doi:10.1038/s41551-024-01296-2

Public

Safer and efficient base editing and prime editing via ribonucleoproteins delivered through optimized lipid-nanoparticle formulations

Published in Nature Biomedical Engineering • Nov 28, 2024

Authors:

Rafał Hołubowicz

Samuel W. Du

Jiin Felgner

Abstract

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...

Subject

Ribonucleoprotein

Guide RNA

Chemistry

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