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Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates.
Costa-Verdera, Helena; Collaud, Fanny; Riling, Christopher R; Sellier, Pauline; Nordin, Jayme M L; Preston, G Michael; Cagin, Umut; Fabregue, Julien; Barral, Simon; Moya-Nilges, Maryse; Krijnse-Locker, Jacomina; van Wittenberghe, Laetitia; Daniele, Natalie; Gjata, Bernard; Cosette, Jeremie; Abad, Catalina; Simon-Sola, Marcelo; Charles, Severine; Li, Mathew; Crosariol, Marco; Antrilli, Tom; Quinn, William J; Gross, David A; Boyer, Olivier; Anguela, Xavier M; Armour, Sean M; Colella, Pasqualina; Ronzitti, Giuseppe; Mingozzi, Federico.
Afiliação
  • Costa-Verdera H; Genethon, 91000, Evry, France.
  • Collaud F; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France.
  • Riling CR; Sorbonne University Paris and INSERM U974, 75013, Paris, France.
  • Sellier P; Genethon, 91000, Evry, France.
  • Nordin JML; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France.
  • Preston GM; Spark Therapeutics, Philadelphia, PA, 19104, USA.
  • Cagin U; Genethon, 91000, Evry, France.
  • Fabregue J; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France.
  • Barral S; Spark Therapeutics, Philadelphia, PA, 19104, USA.
  • Moya-Nilges M; Spark Therapeutics, Philadelphia, PA, 19104, USA.
  • Krijnse-Locker J; Genethon, 91000, Evry, France.
  • van Wittenberghe L; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France.
  • Daniele N; Genethon, 91000, Evry, France.
  • Gjata B; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France.
  • Cosette J; Genethon, 91000, Evry, France.
  • Abad C; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France.
  • Simon-Sola M; Pasteur Institute, 75015, Paris, France.
  • Charles S; Pasteur Institute, 75015, Paris, France.
  • Li M; Genethon, 91000, Evry, France.
  • Crosariol M; Genethon, 91000, Evry, France.
  • Antrilli T; Genethon, 91000, Evry, France.
  • Quinn WJ; Genethon, 91000, Evry, France.
  • Gross DA; Université de Rouen Normandie-IRIB, 76183, Rouen, France.
  • Boyer O; Genethon, 91000, Evry, France.
  • Anguela XM; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France.
  • Armour SM; Genethon, 91000, Evry, France.
  • Colella P; Université Paris-Saclay, Univ Evry, Inserm, Integrare research Unit UMR_S951, 91000, Evry, France.
  • Ronzitti G; Spark Therapeutics, Philadelphia, PA, 19104, USA.
  • Mingozzi F; Spark Therapeutics, Philadelphia, PA, 19104, USA.
Nat Commun ; 12(1): 6393, 2021 11 04.
Article em En | MEDLINE | ID: mdl-34737297
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doença de Depósito de Glicogênio Tipo II / Alfa-Glucosidases Limite: Animals Idioma: En Ano de publicação: 2021 Tipo de documento: Article
Texto completo
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doença de Depósito de Glicogênio Tipo II / Alfa-Glucosidases Limite: Animals Idioma: En Ano de publicação: 2021 Tipo de documento: Article