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

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 homozygous...

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Detalles Bibliográficos
Autores principales: Kasiewicz, Lisa N., Biswas, Souvik, Beach, Aaron, Ren, Huilan, Dutta, Chaitali, Mazzola, Anne Marie, Rohde, Ellen, Chadwick, Alexandra, Cheng, Christopher, Garcia, Sara P., Iyer, Sowmya, Matsumoto, Yuri, Khera, Amit V., Musunuru, Kiran, Kathiresan, Sekar, Malyala, Padma, Rajeev, Kallanthottathil G., Bellinger, Andrew M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10185539/
https://www.ncbi.nlm.nih.gov/pubmed/37188660
http://dx.doi.org/10.1038/s41467-023-37465-1

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