Ontology highlight
ABSTRACT:
SUBMITTER: Soria LR
PROVIDER: S-EPMC7863400 | biostudies-literature | 2021 Feb
REPOSITORIES: biostudies-literature
Soria Leandro R LR Gurung Sonam S De Sabbata Giulia G Perocheau Dany P DP De Angelis Angela A Bruno Gemma G Polishchuk Elena E Paris Debora D Cuomo Paola P Motta Andrea A Orford Michael M Khalil Youssef Y Eaton Simon S Mills Philippa B PB Waddington Simon N SN Settembre Carmine C Muro Andrés F AF Baruteau Julien J Brunetti-Pierri Nicola N
EMBO molecular medicine 20201228 2
Urea cycle disorders (UCD) are inherited defects in clearance of waste nitrogen with high morbidity and mortality. Novel and more effective therapies for UCD are needed. Studies in mice with constitutive activation of autophagy unravelled Beclin-1 as druggable candidate for therapy of hyperammonemia. Next, we investigated efficacy of cell-penetrating autophagy-inducing Tat-Beclin-1 (TB-1) peptide for therapy of the two most common UCD, namely ornithine transcarbamylase (OTC) and argininosuccinat ...[more]