Author:
Cacciante Francesco,Gennaro Mariangela,Sagona Giulia,Mazziotti Raffaele,Lupori Leonardo,Cerri Elisa,Putignano Elena,Butt Mark,Do Minh-Ha T.,McKew John C.,Alessandrì Maria Grazia,Battini Roberta,Cioni Giovanni,Pizzorusso Tommaso,Baroncelli Laura
Abstract
AbstractCreatine Transporter Deficiency (CTD) is an inborn error of metabolism presenting with intellectual disability, behavioral disturbances and epilepsy. There is currently no cure for this disorder. Here, we employed novel biomarkers for monitoring brain function, together with well-established behavioral readouts for CTD mice, to longitudinally study the therapeutic efficacy of cyclocreatine (cCr) at the preclinical level. Our results show that cCr treatment is able to partially correct hemodynamic responses and EEG abnormalities, improve cognitive deficits, revert autistic-like behaviors and protect against seizures. This study provides encouraging data to support the potential therapeutic benefit of cyclocreatine or other chemically modified lipophilic analogs of Cr.
Funder
Lumos Pharma
Italian Ministry of Health
Publisher
Springer Science and Business Media LLC
Cited by
16 articles.
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