GalNAc-Lipid nanoparticles enable non-LDLR dependent hepatic delivery of a CRISPR base editing therapy

Abstract Lipid nanoparticles have demonstrated utility in hepatic delivery of a range of therapeutic modalities and typically deliver their cargo via low-density lipoprotein receptor-mediated endocytosis. For patients lacking sufficient low-density lipoprotein receptor activity, such as those with h...

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Egile Nagusiak:	Lisa N. Kasiewicz, Souvik Biswas, Aaron Beach, Huilan Ren, Chaitali Dutta, Anne Marie Mazzola, Ellen Rohde, Alexandra Chadwick, Christopher Cheng, Sara P. Garcia, Sowmya Iyer, Yuri Matsumoto, Amit V. Khera, Kiran Musunuru, Sekar Kathiresan, Padma Malyala, Kallanthottathil G. Rajeev, Andrew M. Bellinger
Formatua:	Artigo
Hizkuntza:	Inglês
Argitaratua:	Nature Portfolio 2023-05-01
Saila:	Nature Communications
Sarrera elektronikoa:	https://doi.org/10.1038/s41467-023-37465-1
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