Optic neuropathy linked to ACAD9 pathogenic variants: a potentially riboflavin-responsive disorder?
暂无分享,去创建一个
M. Koenig | G. Lenaers | P. Reynier | N. Leboucq | P. Meyer | C. Delettre | N. Gueguen | E. Sarzi | I. Meunier | A. Roubertie | G. Manes | P. Amedro | Mehdi Benkirane | J. Piarroux | Emmanuelle Sarzi
[1] M. Moosajee,et al. From Transcriptomics to Treatment in Inherited Optic Neuropathies , 2021, Genes.
[2] M. Soler‐Lopez,et al. Assembly of The Mitochondrial Complex I Assembly Complex Suggests a Regulatory Role for Deflavination , 2020, Angewandte Chemie.
[3] P. Bross,et al. Riboflavin Deficiency—Implications for General Human Health and Inborn Errors of Metabolism , 2020, International journal of molecular sciences.
[4] M. Lazarou,et al. Dissecting the Roles of Mitochondrial Complex I Intermediate Assembly (MCIA) Complex Factors in the Biogenesis of Complex I , 2019, bioRxiv.
[5] Y. Okazaki,et al. Successful treatment of infantile-onset ACAD9-related cardiomyopathy with a combination of sodium pyruvate, beta-blocker, and coenzyme Q10 , 2019, Journal of pediatric endocrinology & metabolism : JPEM.
[6] L. Sazanov,et al. Mammalian Mitochondrial Complex I Structure and Disease-Causing Mutations. , 2018, Trends in cell biology.
[7] Robert W. Taylor,et al. Clinical, biochemical and genetic spectrum of 70 patients with ACAD9 deficiency: is riboflavin supplementation effective? , 2018, Orphanet Journal of Rare Diseases.
[8] B. Schrank,et al. Lifetime exercise intolerance with lactic acidosis as key manifestation of novel compound heterozygous ACAD9 mutations causing complex I deficiency , 2017, Neuromuscular Disorders.
[9] S. Seneca,et al. Evidence of a wide spectrum of cardiac involvement due to ACAD9 mutations: Report on nine patients. , 2016, Molecular genetics and metabolism.
[10] A. Munnich,et al. High incidence and variable clinical outcome of cardiac hypertrophy due to ACAD9 mutations in childhood , 2015, European Journal of Human Genetics.
[11] A. Laquérriere,et al. Lethal Neonatal Progression of Fetal Cardiomegaly Associated to ACAD9 Deficiency. , 2015, JIMD reports.
[12] Robert W. Taylor,et al. Complex I assembly function and fatty acid oxidation enzyme activity of ACAD9 both contribute to disease severity in ACAD9 deficiency. , 2015, Human molecular genetics.
[13] H. te Brinke,et al. ACAD9, a complex I assembly factor with a moonlighting function in fatty acid oxidation deficiencies. , 2014, Human molecular genetics.
[14] C. La Morgia,et al. The optic nerve: A “mito-window” on mitochondrial neurodegeneration , 2013, Molecular and Cellular Neuroscience.
[15] M. Tarnopolsky,et al. Natural disease course and genotype-phenotype correlations in Complex I deficiency caused by nuclear gene defects: what we learned from 130 cases , 2012, Journal of Inherited Metabolic Disease.
[16] H. Smeets,et al. Riboflavin-responsive oxidative phosphorylation complex I deficiency caused by defective ACAD9: new function for an old gene. , 2011, Brain : a journal of neurology.
[17] Thomas Meitinger,et al. Exome sequencing identifies ACAD9 mutations as a cause of complex I deficiency , 2010, Nature Genetics.
[18] Rutger O. Vogel,et al. Acyl-CoA dehydrogenase 9 is required for the biogenesis of oxidative phosphorylation complex I. , 2010, Cell metabolism.