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
- Fondation Le Jeune
- Lucane Pharma
- X-traordinaire
- NIH [HD080910]
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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