Effective AAV-mediated gene therapy in a mouse model of ethylmalonic encephalopathy
about
AAV-mediated liver-specific MPV17 expression restores mtDNA levels and prevents diet-induced liver failureMitochondrial diseases caused by toxic compound accumulation: from etiopathology to therapeutic approachesToward a therapy for mitochondrial diseaseBrain and muscle redox imbalance elicited by acute ethylmalonic acid administration.Mitochondrial Diseases Part III: Therapeutic interventions in mouse models of OXPHOS deficiencies.Short peptides from leucyl-tRNA synthetase rescue disease-causing mitochondrial tRNA point mutations.C4.4A gene ablation is compatible with normal epidermal development and causes modest overt phenotypes.Emerging therapies for mitochondrial disorders.Proteome adaptations in Ethe1-deficient mice indicate a role in lipid catabolism and cytoskeleton organization via post-translational protein modifications.The clinical maze of mitochondrial neurologyDevelopment of pharmacological strategies for mitochondrial disorders.Altered sulfide (H(2)S) metabolism in ethylmalonic encephalopathy.Gastrointestinal and hepatic manifestations of mitochondrial disorders.Mitochondrial dysfunction in central nervous system white matter disorders.Emerging aspects of treatment in mitochondrial disorders.AAV9-based gene therapy partially ameliorates the clinical phenotype of a mouse model of Leigh syndrome.Gene therapy using a liver-targeted AAV vector restores nucleoside and nucleotide homeostasis in a murine model of MNGIE.Liver transplant in ethylmalonic encephalopathy: a new treatment for an otherwise fatal disease.The isolated carboxy-terminal domain of human mitochondrial leucyl-tRNA synthetase rescues the pathological phenotype of mitochondrial tRNA mutations in human cellsTurkish case of ethylmalonic encephalopathy misdiagnosed as short chain acyl-CoA dehydrogenase deficiency.Ethylmalonic Encephalopathy in an Indian Boy.Towards a therapy for mitochondrial disease: an update
P2860
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P2860
Effective AAV-mediated gene therapy in a mouse model of ethylmalonic encephalopathy
description
2012 nî lūn-bûn
@nan
2012年の論文
@ja
2012年学术文章
@wuu
2012年学术文章
@zh-cn
2012年学术文章
@zh-hans
2012年学术文章
@zh-my
2012年学术文章
@zh-sg
2012年學術文章
@yue
2012年學術文章
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2012年學術文章
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name
Effective AAV-mediated gene therapy in a mouse model of ethylmalonic encephalopathy
@ast
Effective AAV-mediated gene therapy in a mouse model of ethylmalonic encephalopathy
@en
type
label
Effective AAV-mediated gene therapy in a mouse model of ethylmalonic encephalopathy
@ast
Effective AAV-mediated gene therapy in a mouse model of ethylmalonic encephalopathy
@en
prefLabel
Effective AAV-mediated gene therapy in a mouse model of ethylmalonic encephalopathy
@ast
Effective AAV-mediated gene therapy in a mouse model of ethylmalonic encephalopathy
@en
P2093
P2860
P356
P1476
Effective AAV-mediated gene therapy in a mouse model of ethylmalonic encephalopathy
@en
P2093
Alberto Auricchio
Alberto Burlina
Carlo Viscomi
Ivano Di Meo
P2860
P304
P356
10.1002/EMMM.201201433
P577
2012-08-20T00:00:00Z