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In utero CRISPR-mediated therapeutic editing of metabolic genes.
Rossidis, Avery C; Stratigis, John D; Chadwick, Alexandra C; Hartman, Heather A; Ahn, Nicholas J; Li, Haiying; Singh, Kshitiz; Coons, Barbara E; Li, Li; Lv, Wenjian; Zoltick, Philip W; Alapati, Deepthi; Zacharias, William; Jain, Rajan; Morrisey, Edward E; Musunuru, Kiran; Peranteau, William H.
Affiliation
  • Rossidis AC; Division of Pediatric General, Thoracic, and Fetal Surgery, The Center for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Stratigis JD; Division of Pediatric General, Thoracic, and Fetal Surgery, The Center for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Chadwick AC; Department of Medicine, Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.
  • Hartman HA; Department of Genetics, Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.
  • Ahn NJ; Division of Pediatric General, Thoracic, and Fetal Surgery, The Center for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Li H; Division of Pediatric General, Thoracic, and Fetal Surgery, The Center for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Singh K; Division of Pediatric General, Thoracic, and Fetal Surgery, The Center for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Coons BE; Division of Pediatric General, Thoracic, and Fetal Surgery, The Center for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Li L; Division of Pediatric General, Thoracic, and Fetal Surgery, The Center for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Lv W; Department of Medicine, Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.
  • Zoltick PW; Department of Genetics, Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.
  • Alapati D; Department of Medicine, Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.
  • Zacharias W; Department of Genetics, Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.
  • Jain R; Division of Pediatric General, Thoracic, and Fetal Surgery, The Center for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Morrisey EE; Department of Medicine, Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.
  • Musunuru K; Department of Cell and Developmental Biology, Institute for Regenerative Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.
  • Peranteau WH; Department of Medicine, Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.
Nat Med ; 24(10): 1513-1518, 2018 10.
Article in En | MEDLINE | ID: mdl-30297903
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Oxidoreductases / Genetic Therapy / Tyrosinemias / Proprotein Convertase 9 Limits: Animals / Humans Language: En Journal: Nat Med Journal subject: BIOLOGIA MOLECULAR / MEDICINA Year: 2018 Document type: Article Affiliation country: United States Country of publication: United States
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Oxidoreductases / Genetic Therapy / Tyrosinemias / Proprotein Convertase 9 Limits: Animals / Humans Language: En Journal: Nat Med Journal subject: BIOLOGIA MOLECULAR / MEDICINA Year: 2018 Document type: Article Affiliation country: United States Country of publication: United States