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Gene Therapy in Combination with Nitrogen Scavenger Pretreatment Corrects Biochemical and Behavioral Abnormalities of Infant Citrullinemia Type 1 Mice.
Bazo, Andrea; Lantero, Aquilino; Mauleón, Itsaso; Neri, Leire; Poms, Martin; Häberle, Johannes; Ricobaraza, Ana; Bénichou, Bernard; Combal, Jean-Philippe; Gonzalez-Aseguinolaza, Gloria; Aldabe, Rafael.
Afiliação
  • Bazo A; Division of Gene Therapy and Regulation of Gene Expression, CIMA, University of Navarra, 31008 Pamplona, Spain.
  • Lantero A; Vivet Therapeutics, S.L., 31008 Pamplona, Spain.
  • Mauleón I; Division of Gene Therapy and Regulation of Gene Expression, CIMA, University of Navarra, 31008 Pamplona, Spain.
  • Neri L; Vivet Therapeutics, S.L., 31008 Pamplona, Spain.
  • Poms M; Department of Clinical Chemistry and Biochemistry, University Children's Hospital Zurich, University of Zurich, 8091 Zurich, Switzerland.
  • Häberle J; Division of Metabolism, Children's Research Centre (CRC), University Children's Hospital Zurich, 8091 Zurich, Switzerland.
  • Ricobaraza A; Division of Gene Therapy and Regulation of Gene Expression, CIMA, University of Navarra, 31008 Pamplona, Spain.
  • Bénichou B; Vivet Therapeutics, S.A.S., 75008 Paris, France.
  • Combal JP; Vivet Therapeutics, S.A.S., 75008 Paris, France.
  • Gonzalez-Aseguinolaza G; Division of Gene Therapy and Regulation of Gene Expression, CIMA, University of Navarra, 31008 Pamplona, Spain.
  • Aldabe R; Vivet Therapeutics, S.L., 31008 Pamplona, Spain.
Int J Mol Sci ; 23(23)2022 Nov 29.
Article em En | MEDLINE | ID: mdl-36499263

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Citrulinemia / Amônia Limite: Animals Idioma: En Ano de publicação: 2022 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Citrulinemia / Amônia Limite: Animals Idioma: En Ano de publicação: 2022 Tipo de documento: Article