Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector.

De Sabbata, Giulia; Boisgerault, Florence; Guarnaccia, Corrado; Iaconcig, Alessandra; Bortolussi, Giulia; Collaud, Fanny; Ronzitti, Giuseppe; Sola, Marcelo Simon; Vidal, Patrice; Rouillon, Jeremy; Charles, Severine; Nicastro, Emanuele; D'Antiga, Lorenzo; Ilyinskii, Petr; Mingozzi, Federico; Kishimoto, Takashi Kei; Muro, Andrés F

De Sabbata, Giulia; Boisgerault, Florence; Guarnaccia, Corrado; Iaconcig, Alessandra; Bortolussi, Giulia; Collaud, Fanny; Ronzitti, Giuseppe; Sola, Marcelo Simon; Vidal, Patrice; Rouillon, Jeremy; Charles, Severine; Nicastro, Emanuele; D'Antiga, Lorenzo; Ilyinskii, Petr; Mingozzi, Federico; Kishimoto, Takashi Kei; Muro, Andrés F.

Afiliación

De Sabbata G; International Center for Genetic Engineering and Biotechnology (ICGEB), 34149 Trieste, Italy.
Boisgerault F; Généthon, 91000 Evry, France.
Guarnaccia C; Université Paris-Saclay, Université Evry, INSERM, Généthon, Integrare Research Unit UMR_S951, 91000 Evry, France.
Iaconcig A; International Center for Genetic Engineering and Biotechnology (ICGEB), 34149 Trieste, Italy.
Bortolussi G; International Center for Genetic Engineering and Biotechnology (ICGEB), 34149 Trieste, Italy.
Collaud F; International Center for Genetic Engineering and Biotechnology (ICGEB), 34149 Trieste, Italy.
Ronzitti G; Généthon, 91000 Evry, France.
Sola MS; Université Paris-Saclay, Université Evry, INSERM, Généthon, Integrare Research Unit UMR_S951, 91000 Evry, France.
Vidal P; Généthon, 91000 Evry, France.
Rouillon J; Université Paris-Saclay, Université Evry, INSERM, Généthon, Integrare Research Unit UMR_S951, 91000 Evry, France.
Charles S; Généthon, 91000 Evry, France.
Nicastro E; Université Paris-Saclay, Université Evry, INSERM, Généthon, Integrare Research Unit UMR_S951, 91000 Evry, France.
D'Antiga L; Généthon, 91000 Evry, France.
Ilyinskii P; Université Paris-Saclay, Université Evry, INSERM, Généthon, Integrare Research Unit UMR_S951, 91000 Evry, France.
Mingozzi F; Généthon, 91000 Evry, France.
Kishimoto TK; Université Paris-Saclay, Université Evry, INSERM, Généthon, Integrare Research Unit UMR_S951, 91000 Evry, France.
Muro AF; Généthon, 91000 Evry, France.

Mol Ther Methods Clin Dev ; 20: 169-180, 2021 Mar 12.

Article en En | MEDLINE | ID: mdl-33473356

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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: Mol Ther Methods Clin Dev Año: 2021 Tipo del documento: Article País de afiliación: Italia Pais de publicación: Estados Unidos

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