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

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.

SUBMITTER: Ullio-Gamboa G 

PROVIDER: S-EPMC6613044 | biostudies-literature | 2019 Jun

REPOSITORIES: biostudies-literature