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An Exon-Specific Small Nuclear U1 RNA (ExSpeU1) Improves Hepatic OTC Expression in a Splicing-Defective spf/ash Mouse Model of Ornithine Transcarbamylase Deficiency.
Balestra, Dario; Ferrarese, Mattia; Lombardi, Silvia; Ziliotto, Nicole; Branchini, Alessio; Petersen, Naomi; Bosma, Piter; Pinotti, Mirko; van de Graaf, Stan F J.
Afiliación
  • Balestra D; Department of Life Sciences and Biotechnology and LTTA, University of Ferrara, 44121 Ferrara, Italy.
  • Ferrarese M; Department of Life Sciences and Biotechnology and LTTA, University of Ferrara, 44121 Ferrara, Italy.
  • Lombardi S; Department of Life Sciences and Biotechnology and LTTA, University of Ferrara, 44121 Ferrara, Italy.
  • Ziliotto N; Department of Life Sciences and Biotechnology and LTTA, University of Ferrara, 44121 Ferrara, Italy.
  • Branchini A; School of Medicine and Surgery, University of Milano-Bicocca, 20900 Milan, Italy.
  • Petersen N; Department of Life Sciences and Biotechnology and LTTA, University of Ferrara, 44121 Ferrara, Italy.
  • Bosma P; Tytgat Institute for Liver and Intestinal Research and Department of Gastroenterology and Hepatology, Amsterdam UMC, University of Amsterdam, 1105 AZ Amsterdam, The Netherlands.
  • Pinotti M; Tytgat Institute for Liver and Intestinal Research and Department of Gastroenterology and Hepatology, Amsterdam UMC, University of Amsterdam, 1105 AZ Amsterdam, The Netherlands.
  • van de Graaf SFJ; Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam UMC, 1105 AZ Amsterdam, The Netherlands.
Int J Mol Sci ; 21(22)2020 Nov 19.
Article en En | MEDLINE | ID: mdl-33228018
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Ornitina Carbamoiltransferasa / ARN Nuclear Pequeño / Empalme del ARN / Dependovirus / Enfermedad por Deficiencia de Ornitina Carbamoiltransferasa Límite: Animals / Humans / Male Idioma: En Revista: Int J Mol Sci Año: 2020 Tipo del documento: Article País de afiliación: Italia Pais de publicación: Suiza
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Ornitina Carbamoiltransferasa / ARN Nuclear Pequeño / Empalme del ARN / Dependovirus / Enfermedad por Deficiencia de Ornitina Carbamoiltransferasa Límite: Animals / Humans / Male Idioma: En Revista: Int J Mol Sci Año: 2020 Tipo del documento: Article País de afiliación: Italia Pais de publicación: Suiza