Branched endosomal disruptor (BEND) lipids mediate delivery of mRNA and CRISPR-Cas9 ribonucleoprotein complex for hepatic gene editing and T cell engineering

Branched endosomal disruptor (BEND) lipids mediate delivery of mRNA and CRISPR-Cas9 ribonucleoprotein complex for hepatic gene editing and T cell engineering

Abstract Lipid nanoparticles (LNPs) are the preeminent non-viral drug delivery vehicle for mRNA-based therapies. Immense effort has been placed on optimizing the ionizable lipid (IL) structure, which contains an amine core conjugated to lipid tails, as small molecular adjustments can result in subst...

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Main Authors: Marshall S. Padilla, Kaitlin Mrksich, Yiming Wang, Rebecca M. Haley, Jacqueline J. Li, Emily L. Han, Rakan El-Mayta, Emily H. Kim, Sofia Dias, Ningqiang Gong, Sridatta V. Teerdhala, Xuexiang Han, Vivek Chowdhary, Lulu Xue, Zain Siddiqui, Hannah M. Yamagata, Dongyoon Kim, Il-Chul Yoon, James M. Wilson, Ravi Radhakrishnan, Michael J. Mitchell
Format: Article
Language:English
Published: Nature Portfolio 2025-01-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-024-55137-6
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https://doi.org/10.1038/s41467-024-55137-6

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