Dodecyl creatine ester-loaded nanoemulsion as a promising therapy for creatine transporter deficiency-Reference-Cited by-同舟云学术

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

Published:2019-06 Issue:12 Volume:14 Page:1579-1593
ISSN:1743-5889
Container-title:Nanomedicine
language:en
Short-container-title:Nanomedicine

Author:

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

Affiliation:

1. Service de Pharmacologie et d’Immunoanalyse, CEA, Université Paris-Saclay, F-91191 Gif-sur-Yvette, 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

4. Service de Pharmacologie et d’Immunoanalyse (SPI), Plateforme Smart-MS, CEA, INRA, Université Paris-Saclay, F-91191 Gif-sur-Yvette, 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

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

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.

Publisher

Future Medicine Ltd

Subject

Development,General Materials Science,Biomedical Engineering,Medicine (miscellaneous),Bioengineering

Link

https://www.futuremedicine.com/doi/pdf/10.2217/nnm-2019-0059

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