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

十二烷基肌酸酯纳米乳剂有望成为治疗肌酸转运蛋白缺乏症的良药

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作者:Gabriela Ullio-Gamboa, Kenea C Udobi, Sophie Dezard, Marla K Perna, Keila N Miles, Narciso Costa, Frédéric Taran, Alain Pruvost, Jean-Pierre Benoit, Matthew R Skelton, Pascale de Lonlay, Aloïse Mabondzo

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 &

Conclusion

These results highlight the potential value of DCE-ME as promising therapy for creatine transporter deficiency.

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.

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