4.7 Article

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

期刊

NANOMEDICINE
卷 14, 期 12, 页码 1579-1593

出版社

FUTURE MEDICINE LTD
DOI: 10.2217/nnm-2019-0059

关键词

creatine; creatine transporter deficiency; dodecyl creatine ester; drug delivery; microemulsion; nasal administration; neurotherapeutics for CTD

资金

  1. Fondation Le Jeune
  2. Lucane Pharma
  3. X-traordinaire
  4. NIH [HD080910]

向作者/读者索取更多资源

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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