TY - GEN AU - Hidalgo Gutiérrez, Agustín AU - Barriocanal Casado, Eliana AU - Bakkali, Mohammed AU - Díaz-Casado, Elena AU - Sánchez-Maldonado, Laura AU - Romero Pérez, Miguel AU - Sayed, Ramy K. A. AU - Prehn, Cornelia AU - Escames Rosa, Germaine AU - Duarte Pérez, Juan Manuel AU - Acuña Castroviejo, Darío AU - López García, Luis Carlos PY - 2018 UR - http://hdl.handle.net/10481/53973 AB - Coenzyme Q (CoQ) deficiency has been associated with primary defects in the CoQ biosynthetic pathway or to secondary events. In some cases, the exogenous CoQ supplementation has limited efficacy. In the Coq9R239X mouse model with fatal... LA - eng PB - European Molecular Biology Organization (EMBO) KW - Genetics KW - Gene Therapy & Genetic Disease KW - Pharmacology & Drug Discovery TI - β-RA reduces DMQ/CoQ ratio and rescues the encephalopathic phenotype in Coq9R239X mice DO - 10.15252/emmm.201809466 ER -