In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels
In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels
About this item
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Author / Creator
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. and Schwank, Gerald
Publisher
New York: Nature Publishing Group US
Journal title
Language
English
Formats
Publication information
Publisher
New York: Nature Publishing Group US
Subjects
More information
Scope and Contents
Contents
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 (LDL) levels. Lipid nanoparticle–based delivery o...
Alternative Titles
Full title
In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels
Authors, Artists and Contributors
Author / Creator
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
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_8352781
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_8352781
Other Identifiers
ISSN
1087-0156
E-ISSN
1546-1696
DOI
10.1038/s41587-021-00933-4
