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Dodecyl creatine ester-loaded nanoemulsion as a promising therapy for creatine transporter deficiency.

Authors
  • Ullio-Gamboa, Gabriela1
  • Udobi, Kenea C2
  • Dezard, Sophie3
  • Perna, Marla K2
  • Miles, Keila N2
  • Costa, Narciso1
  • Taran, Frédéric3
  • Pruvost, Alain4
  • Benoit, Jean-Pierre5
  • Skelton, Matthew R2
  • Lonlay, Pascale de6
  • Mabondzo, Aloïse1
  • 1 Service de Pharmacologie et d'Immunoanalyse, CEA, Université Paris-Saclay, F-91191 Gif-sur-Yvette, France. , (France)
  • 2 Department of Pediatrics, University of Cincinnati College of Medicine & Division of Neurology, Cincinnati Children's Research Foundation, Cincinnati, OH, USA.
  • 3 Service de Chimie Bio Organique et de Marquage CEA, Université Paris-Saclay, F-91191 Gif-sur-Yvette, France. , (France)
  • 4 Service de Pharmacologie et d'Immunoanalyse (SPI), Plateforme Smart-MS, CEA, INRA, Université Paris-Saclay, F-91191 Gif-sur-Yvette, France. , (France)
  • 5 LUNAM Université-Micro et Nanomédecines Biomimétiques, F-49933 Angers, France, INSERM U1066, IBS-CHU, 4 rue Larrey, F-49933 Angers Cedex 9, France. , (France)
  • 6 Centre de Référence des Maladies Héréditaires du Métabolisme de l'Enfant et de l'Adulte, Hôpital Necker-Enfants Malades, APHP, Université Paris Descartes, Imagine, INEM, Filière G2M, metabERN, Paris, France. , (France)
Type
Published Article
Journal
Nanomedicine
Publisher
Future Medicine
Publication Date
Jun 01, 2019
Volume
14
Issue
12
Pages
1579–1593
Identifiers
DOI: 10.2217/nnm-2019-0059
PMID: 31038003
Source
Medline
Keywords
Language
English
License
Unknown

Abstract

Creatine transporter (CrT) deficiency is an X-linked intellectual disability caused by mutations of CrT. Aim: This work focus on the preclinical development of a new therapeutic approach based on a microemulsion (ME) as drug delivery system for dodecyl creatine ester (DCE). Materials & methods: DCE-ME was prepared by titration method. Novel object recognition (NOR) tests were performed before and after DCE-ME treatment on Slc6a8-/y mice. Results: Intranasal administration with DCE-ME improved NOR performance in Slc6a8-/y mice. Slc6a8-/y mice treated with DCE-ME had increased striatal ATP levels mainly in the striatum compared with vehicle-treated Slc6a8-/y mice which was associated with increased expression of synaptic markers. Conclusion: These results highlight the potential value of DCE-ME as promising therapy for creatine transporter deficiency.

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