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

Autor: Rothgangl T; University of Zurich, Institute for Pharmacology and Toxicology, Zurich, Switzerland., Dennis MK; Acuitas Therapeutics Inc., Vancouver, BC, Canada., Lin PJC; Acuitas Therapeutics Inc., Vancouver, BC, Canada., Oka R; Oncode Institute, Princess Máxima Center for Pediatric Oncology, Utrecht, Netherlands., Witzigmann D; University of Zurich, Institute for Pharmacology and Toxicology, Zurich, Switzerland., Villiger L; University of Zurich, Institute for Pharmacology and Toxicology, Zurich, Switzerland., Qi W; Functional Genomics Center Zurich, ETH Zurich/University of Zurich, Zurich, Switzerland., Hruzova M; Department of Biology, Institute for Molecular Health Sciences, ETH Zurich, Zurich, Switzerland., Kissling L; University of Zurich, Institute for Pharmacology and Toxicology, Zurich, Switzerland., Lenggenhager D; Department of Pathology and Molecular Pathology, University Hospital and University of Zurich, Zurich, Switzerland., Borrelli C; Department of Biosystems Science and Engineering, ETH Zurich, Zurich, Switzerland., Egli S; University of Zurich, Institute for Pharmacology and Toxicology, Zurich, Switzerland., Frey N; Department of Biology, Institute for Molecular Health Sciences, ETH Zurich, Zurich, Switzerland., Bakker N; University of Zurich, Institute for Pharmacology and Toxicology, Zurich, Switzerland., Walker JA 2nd; Synthego Corporation, Redwood City, CA, USA., Kadina AP; Synthego Corporation, Redwood City, CA, USA., Victorov DV; Synthego Corporation, Redwood City, CA, USA., Pacesa M; Department of Biochemistry, University of Zurich, Zurich, Switzerland., Kreutzer S; Functional Genomics Center Zurich, ETH Zurich/University of Zurich, Zurich, Switzerland.; Genome Engineering and Measurement Laboratory, ETH Zurich, Zurich, Switzerland., Kontarakis Z; Functional Genomics Center Zurich, ETH Zurich/University of Zurich, Zurich, Switzerland.; Genome Engineering and Measurement Laboratory, ETH Zurich, Zurich, Switzerland., Moor A; Department of Biosystems Science and Engineering, ETH Zurich, Zurich, Switzerland., Jinek M; Department of Biochemistry, University of Zurich, Zurich, Switzerland., Weissman D; Department of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Stoffel M; Department of Biology, Institute for Molecular Health Sciences, ETH Zurich, Zurich, Switzerland., van Boxtel R; Oncode Institute, Princess Máxima Center for Pediatric Oncology, Utrecht, Netherlands., Holden K; Synthego Corporation, Redwood City, CA, USA., Pardi N; Department of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Thöny B; Division of Metabolism and Children's Research Centre, University Children's Hospital Zurich, Zurich, Switzerland.; Zurich Center for Integrative Human Physiology, Zurich, Switzerland.; Neuroscience Center Zurich, Zurich, Switzerland., Häberle J; Division of Metabolism and Children's Research Centre, University Children's Hospital Zurich, Zurich, Switzerland.; Zurich Center for Integrative Human Physiology, Zurich, Switzerland.; Institute of Molecular Life Sciences, University of Zurich, Zurich, Switzerland., Tam YK; Acuitas Therapeutics Inc., Vancouver, BC, Canada., Semple SC; Acuitas Therapeutics Inc., Vancouver, BC, Canada. ssemple@acuitastx.com., Schwank G; University of Zurich, Institute for Pharmacology and Toxicology, Zurich, Switzerland. schwank@pharma.uzh.ch.; Department of Biology, Institute for Molecular Health Sciences, ETH Zurich, Zurich, Switzerland. schwank@pharma.uzh.ch.

Publikováno v: Nature biotechnology [Nat Biotechnol] 2021 Aug; Vol. 39 (8), pp. 949-957. Date of Electronic Publication: 2021 May 19.