In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels

Most known pathogenic point mutations in humans are C•G to T•A substitutions, which can be directly repaired by adenine base editors (ABEs). In this study, we investigated the efficacy and safety of ABEs in the livers of mice and cynomolgus macaques for the reduction of blood low-density lipoprotein...

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Detalles Bibliográficos
Autores principales: Rothgangl, Tanja, Dennis, Melissa K., Lin, Paulo J. C., Oka, Rurika, Witzigmann, Dominik, Villiger, Lukas, Qi, Weihong, Hruzova, Martina, Kissling, Lucas, Lenggenhager, Daniela, Borrelli, Costanza, Egli, Sabina, Frey, Nina, Bakker, Noëlle, Walker, John A., Kadina, Anastasia P., Victorov, Denis V., Pacesa, Martin, Kreutzer, Susanne, Kontarakis, Zacharias, Moor, Andreas, Jinek, Martin, Weissman, Drew, Stoffel, Markus, van Boxtel, Ruben, Holden, Kevin, Pardi, Norbert, Thöny, Beat, Häberle, Johannes, Tam, Ying K., Semple, Sean C., Schwank, Gerald
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group US 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8352781/
https://www.ncbi.nlm.nih.gov/pubmed/34012094
http://dx.doi.org/10.1038/s41587-021-00933-4

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