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In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates.

Autor: Musunuru K; Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.; Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.; Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA., Chadwick AC; Verve Therapeutics, Cambridge, MA, USA., Mizoguchi T; Verve Therapeutics, Cambridge, MA, USA., Garcia SP; Verve Therapeutics, Cambridge, MA, USA., DeNizio JE; Verve Therapeutics, Cambridge, MA, USA., Reiss CW; Verve Therapeutics, Cambridge, MA, USA., Wang K; Verve Therapeutics, Cambridge, MA, USA., Iyer S; Verve Therapeutics, Cambridge, MA, USA., Dutta C; Verve Therapeutics, Cambridge, MA, USA., Clendaniel V; Verve Therapeutics, Cambridge, MA, USA., Amaonye M; Verve Therapeutics, Cambridge, MA, USA., Beach A; Verve Therapeutics, Cambridge, MA, USA., Berth K; Verve Therapeutics, Cambridge, MA, USA., Biswas S; Verve Therapeutics, Cambridge, MA, USA., Braun MC; Verve Therapeutics, Cambridge, MA, USA., Chen HM; Verve Therapeutics, Cambridge, MA, USA., Colace TV; Verve Therapeutics, Cambridge, MA, USA., Ganey JD; Verve Therapeutics, Cambridge, MA, USA., Gangopadhyay SA; Verve Therapeutics, Cambridge, MA, USA., Garrity R; Verve Therapeutics, Cambridge, MA, USA., Kasiewicz LN; Verve Therapeutics, Cambridge, MA, USA., Lavoie J; Verve Therapeutics, Cambridge, MA, USA., Madsen JA; Verve Therapeutics, Cambridge, MA, USA., Matsumoto Y; Verve Therapeutics, Cambridge, MA, USA., Mazzola AM; Verve Therapeutics, Cambridge, MA, USA., Nasrullah YS; Verve Therapeutics, Cambridge, MA, USA., Nneji J; Verve Therapeutics, Cambridge, MA, USA., Ren H; Verve Therapeutics, Cambridge, MA, USA., Sanjeev A; Verve Therapeutics, Cambridge, MA, USA., Shay M; Verve Therapeutics, Cambridge, MA, USA., Stahley MR; Verve Therapeutics, Cambridge, MA, USA., Fan SHY; Acuitas Therapeutics, Vancouver, British Columbia, Canada., Tam YK; Acuitas Therapeutics, Vancouver, British Columbia, Canada., Gaudelli NM; Beam Therapeutics, Cambridge, MA, USA., Ciaramella G; Beam Therapeutics, Cambridge, MA, USA., Stolz LE; Verve Therapeutics, Cambridge, MA, USA., Malyala P; Verve Therapeutics, Cambridge, MA, USA., Cheng CJ; Verve Therapeutics, Cambridge, MA, USA., Rajeev KG; Verve Therapeutics, Cambridge, MA, USA., Rohde E; Verve Therapeutics, Cambridge, MA, USA., Bellinger AM; Verve Therapeutics, Cambridge, MA, USA., Kathiresan S; Verve Therapeutics, Cambridge, MA, USA. skathiresan@vervetx.com.

Publikováno v: Nature [Nature] 2021 May; Vol. 593 (7859), pp. 429-434. Date of Electronic Publication: 2021 May 19.

In utero gene editing for monogenic lung disease.

Autor: Alapati D; Department of Pediatrics Nemours, Alfred I duPont Hospital for Children, Wilmington, DE 19803, USA.; Department of Pediatrics Sidney Kimmel Medical College, Thomas Jefferson University Philadelphia, Philadelphia, PA 19107, USA.; Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.; Penn Center for Pulmonary Biology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.; Penn Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania Philadelphia, Philadelphia, PA 19104, USA., Zacharias WJ; Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.; Penn Center for Pulmonary Biology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.; Penn Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania Philadelphia, Philadelphia, PA 19104, USA.; Division of Pulmonary Biology Cincinnati Children's Hospital, Department of Pediatrics University of Cincinnati College of Medicine, Cincinnati, OH 45529, USA., Hartman HA; Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Rossidis AC; Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Stratigis JD; Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Ahn NJ; Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Coons B; Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Zhou S; Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.; Penn Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania Philadelphia, Philadelphia, PA 19104, USA., Li H; Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Singh K; Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Katzen J; Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.; Penn Center for Pulmonary Biology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA., Tomer Y; Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.; Penn Center for Pulmonary Biology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA., Chadwick AC; Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.; Penn Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania Philadelphia, Philadelphia, PA 19104, USA., Musunuru K; Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.; Penn Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania Philadelphia, Philadelphia, PA 19104, USA., Beers MF; Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.; Penn Center for Pulmonary Biology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA., Morrisey EE; Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA. emorrise@pennmedicine.upenn.edu peranteauw@email.chop.edu.; Penn Center for Pulmonary Biology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.; Penn Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania Philadelphia, Philadelphia, PA 19104, USA., Peranteau WH; Center for Fetal Research, Division of General, Thoracic, and Fetal Surgery, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA. emorrise@pennmedicine.upenn.edu peranteauw@email.chop.edu.

Publikováno v: Science translational medicine [Sci Transl Med] 2019 Apr 17; Vol. 11 (488).

Beyond scattering and absorption: Perceptual unmixing of translucent liquids.

Autor: Chadwick AC; Department of Psychology, University of Durham, Durham University Science Site, Durham, UK., Cox G; Department of Experimental Psychology, University of Oxford, Oxford, UK., Smithson HE; Department of Experimental Psychology, University of Oxford, Oxford, UK., Kentridge RW; Department of Psychology, University of Durham, Durham University Science Site, Durham, UK.

Publikováno v: Journal of vision [J Vis] 2018 Oct 01; Vol. 18 (11), pp. 18.

CRISPR-Cas9 Genome Editing for Treatment of Atherogenic Dyslipidemia.

Autor: Chadwick AC; From the Department of Medicine and Department of Genetics, Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia., Musunuru K; From the Department of Medicine and Department of Genetics, Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia. kiranmusunuru@gmail.com.

Publikováno v: Arteriosclerosis, thrombosis, and vascular biology [Arterioscler Thromb Vasc Biol] 2018 Jan; Vol. 38 (1), pp. 12-18. Date of Electronic Publication: 2017 Aug 24.