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Evaluation of the efficacy of cystinosin supplementation through CTNS mRNA delivery in experimental models for cystinosis.
Bondue, Tjessa; Berlingerio, Sante Princiero; Siegerist, Florian; Sendino-Garví, Elena; Schindler, Maximilian; Baelde, Hans Jacobus; Cairoli, Sara; Goffredo, Bianca Maria; Arcolino, Fanny Oliveira; Dieker, Jürgen; Janssen, Manoe Jacoba; Endlich, Nicole; Brock, Roland; Gijsbers, Rik; van den Heuvel, Lambertus; Levtchenko, Elena.
Afiliación
  • Bondue T; Laboratory of Pediatric Nephrology, Department of Development and Regeneration, KU Leuven, Leuven, Belgium.
  • Berlingerio SP; Laboratory of Pediatric Nephrology, Department of Development and Regeneration, KU Leuven, Leuven, Belgium.
  • Siegerist F; Institute of Anatomy and Cell Biology, Universitätsmedizin Greifswald, Greifswald, Germany.
  • Sendino-Garví E; Division Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Utrecht, The Netherlands.
  • Schindler M; Institute of Anatomy and Cell Biology, Universitätsmedizin Greifswald, Greifswald, Germany.
  • Baelde HJ; Department of Pathology, Leiden University Medical Center, Leiden, The Netherlands.
  • Cairoli S; Laboratory of Metabolic Biochemistry, Department of Pediatric Medicine, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
  • Goffredo BM; Laboratory of Metabolic Biochemistry, Department of Pediatric Medicine, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
  • Arcolino FO; Laboratory of Pediatric Nephrology, Department of Development and Regeneration, KU Leuven, Leuven, Belgium.
  • Dieker J; Department of Pediatric Nephrology, Emma Children's Hospital and Emma Center for Personalized Medicine, Amsterdam UMC, Amsterdam, The Netherlands.
  • Janssen MJ; Mercurna B.V., Oss, The Netherlands.
  • Endlich N; Division Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Utrecht, The Netherlands.
  • Brock R; Institute of Anatomy and Cell Biology, Universitätsmedizin Greifswald, Greifswald, Germany.
  • Gijsbers R; Department of Medical Biosciences, Radboud University Medical Center, Nijmegen, The Netherlands.
  • van den Heuvel L; Department of Medical Biochemistry, College of Medicine and Medical Sciences, Arabian Gulf University, Manama, Kingdom of Bahrain.
  • Levtchenko E; Laboratory for Molecular Virology and Gene Therapy, Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium.
Sci Rep ; 13(1): 20961, 2023 11 28.
Article en En | MEDLINE | ID: mdl-38016974
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Texto completo: 1 Bases de datos: MEDLINE Asunto principal: Cistinosis / Sistemas de Transporte de Aminoácidos Neutros Idioma: En Revista: Sci Rep Año: 2023 Tipo del documento: Article País de afiliación: Bélgica
Texto completo
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Texto completo: 1 Bases de datos: MEDLINE Asunto principal: Cistinosis / Sistemas de Transporte de Aminoácidos Neutros Idioma: En Revista: Sci Rep Año: 2023 Tipo del documento: Article País de afiliación: Bélgica