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

Creatine transporter (CrT) deficiency is an X-linked intellectual disability caused by mutations of CrT. 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). DCE-ME was prepared by titration method. Novel object recognition (NOR) tests were performed before and after DCE-ME treatment on mice. Intranasal administration with DCE-ME improved NOR performance in mice. mice treated with DCE-ME had increased striatal ATP levels mainly in the striatum compared with vehicle-treated mice which was associated with increased expression of synaptic markers. These results highlight the potential value of DCE-ME as promising therapy for creatine transporter deficiency.