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Rapid and definitive treatment of phenylketonuria in variant-humanized mice with corrective editing.
Brooks, Dominique L; Carrasco, Manuel J; Qu, Ping; Peranteau, William H; Ahrens-Nicklas, Rebecca C; Musunuru, Kiran; Alameh, Mohamad-Gabriel; Wang, Xiao.
Afiliação
  • Brooks DL; Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.
  • Carrasco MJ; Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.
  • Qu P; Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.
  • Peranteau WH; Department of Bioengineering, George Mason University, Fairfax, Virginia, USA.
  • Ahrens-Nicklas RC; Cardiovascular Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.
  • Musunuru K; Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.
  • Alameh MG; Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.
  • Wang X; The Center for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.
Nat Commun ; 14(1): 3451, 2023 06 10.
Article em En | MEDLINE | ID: mdl-37301931
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Fenilalanina Hidroxilase / Fenilcetonúrias Limite: Animals Idioma: En Ano de publicação: 2023 Tipo de documento: Article
Texto completo
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XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Fenilalanina Hidroxilase / Fenilcetonúrias Limite: Animals Idioma: En Ano de publicação: 2023 Tipo de documento: Article