Treatment of glutaric aciduria type I (GA-I) via intracerebroventricular delivery of GCDH

Autor: Lu Guo, Zhikun Li, Yuhuan Li, Bin Qu, Guanyi Jiao, Chen Liang, Zongbao Lu, Xin-Ge Wang, Cheng Huang, Hongwei Du, Jianmin Liang, Qi Zhou, Wei Li

Publikováno v: Fundamental Research, Vol 2, Iss 6, Pp 836-842 (2022)

Glutaric aciduria type I (GA-I) is an autosomal recessive genetic disorder caused by a deficiency in glutaryl-CoA dehydrogenase (GCDH). Patients who do not receive proper treatment may die from acute encephalopathic crisis. Current treatments for GA-

Externí odkaz: https://doaj.org/article/134907e6088442b3abdf6c19065fb784

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Deglutarylation of glutaryl-CoA dehydrogenase by deacylating enzyme SIRT5 promotes lysine oxidation in mice.

Autor: Bhatt DP; Duke Molecular Physiology Institute, Duke University School of Medicine, Durham, North Carolina, USA; Department of Cell Biology and Physiology, Washington University in St. Louis, St. Louis, Missouri, USA., Mills CA; Duke Molecular Physiology Institute, Duke University School of Medicine, Durham, North Carolina, USA., Anderson KA; Duke Molecular Physiology Institute, Duke University School of Medicine, Durham, North Carolina, USA; Sarah W. Stedman Nutrition and Metabolism Center, Duke University School of Medicine, Durham, North Carolina, USA; Departments of Medicine and Pharmacology & Cancer Biology, Duke University School of Medicine, Durham, North Carolina, USA., Henriques BJ; Biosystems and Integrative Sciences Institute, Faculdade de Ciências, Universidade de Lisboa, Lisboa, Portugal; Departmento de Química e Bioquimica, Faculdade de Ciências, Universidade de Lisboa, Lisboa, Portugal., Lucas TG; Biosystems and Integrative Sciences Institute, Faculdade de Ciências, Universidade de Lisboa, Lisboa, Portugal; Departmento de Química e Bioquimica, Faculdade de Ciências, Universidade de Lisboa, Lisboa, Portugal., Francisco S; Biosystems and Integrative Sciences Institute, Faculdade de Ciências, Universidade de Lisboa, Lisboa, Portugal; Departmento de Química e Bioquimica, Faculdade de Ciências, Universidade de Lisboa, Lisboa, Portugal., Liu J; Departments of Medicine and Pharmacology & Cancer Biology, Duke University School of Medicine, Durham, North Carolina, USA., Ilkayeva OR; Duke Molecular Physiology Institute, Duke University School of Medicine, Durham, North Carolina, USA; Department of Medicine, Division of Endocrinology, Metabolism, and Nutrition, Duke University School of Medicine, Durham, North Carolina, USA., Adams AE; Duke Molecular Physiology Institute, Duke University School of Medicine, Durham, North Carolina, USA., Kulkarni SR; Duke Molecular Physiology Institute, Duke University School of Medicine, Durham, North Carolina, USA., Backos DS; Computational Chemistry and Biology Core Facility, Skaggs School of Pharmacy and Pharmaceutical Sciences, University of Colorado Anschutz Medical Campus, Aurora, Colorado, USA., Major MB; Department of Cell Biology and Physiology, Washington University in St. Louis, St. Louis, Missouri, USA., Grimsrud PA; Duke Molecular Physiology Institute, Duke University School of Medicine, Durham, North Carolina, USA; Department of Medicine, Division of Endocrinology, Metabolism, and Nutrition, Duke University School of Medicine, Durham, North Carolina, USA., Gomes CM; Biosystems and Integrative Sciences Institute, Faculdade de Ciências, Universidade de Lisboa, Lisboa, Portugal; Departmento de Química e Bioquimica, Faculdade de Ciências, Universidade de Lisboa, Lisboa, Portugal., Hirschey MD; Duke Molecular Physiology Institute, Duke University School of Medicine, Durham, North Carolina, USA; Sarah W. Stedman Nutrition and Metabolism Center, Duke University School of Medicine, Durham, North Carolina, USA; Departments of Medicine and Pharmacology & Cancer Biology, Duke University School of Medicine, Durham, North Carolina, USA; Department of Medicine, Division of Endocrinology, Metabolism, and Nutrition, Duke University School of Medicine, Durham, North Carolina, USA. Electronic address: matthew.hirschey@duke.edu.

Publikováno v: The Journal of biological chemistry [J Biol Chem] 2022 Apr; Vol. 298 (4), pp. 101723. Date of Electronic Publication: 2022 Feb 12.