Dodecyl Creatine Ester-Loaded Nanoemulsion as a Promising Therapy for Creatine Transporter Deficiency
Intranasal administration with DCE-ME improved NOR performance in Slc6a8-/y mice and increased striatal ATP levels mainly in the striatum compared with vehicle-treated Slc7-/Y mice, highlighting the potential value of D CE-ME as promising therapy for creatine transporter deficiency.
Abstract
Creatine transporter (CrT) deficiency is an X-linked intellectual disability caused by mutations of CrT. <b>Aim:</b> 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). <b>Materials & methods:</b> DCE-ME was prepared by titration method. Novel object recognition (NOR) tests were performed before and after DCE-ME treatment on <i>Slc6a8<sup>-/y</sup></i> mice. <b>Results:</b> Intranasal administration with DCE-ME improved NOR performance in <i>Slc6a8<sup>-/y</sup></i> mice. <i>Slc6a8<sup>-/y</sup></i> mice treated with DCE-ME had increased striatal ATP levels mainly in the striatum compared with vehicle-treated <i>Slc6a8<sup>-/y</sup></i> mice which was associated with increased expression of synaptic markers. <b>Conclusion:</b> These results highlight the potential value of DCE-ME as promising therapy for creatine transporter deficiency.