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Effective AAV‐mediated gene therapy in a mouse model of ...
Ethylmalonic encephalopathy (EE) is an invariably fatal disease, characterized by the accumulation of hydrogen sulfide (H2S), a highly toxic compound. ETHE1 , encoding sulfur dioxygenase (SDO), which takes part in the mitochondrial pathway that converts sulfide into harmless sulfate, is mutated in EE.
Effective AAV-mediated gene therapy in a mouse model of ...
Effective AAV-mediated gene therapy in a mouse model of ethylmalonic encephalopathy. Ivano Di Meo 1, Alberto ... Massimo Zeviani, Tel: +39 02 2394 2630; Fax: +39 02 23942619. ... d'Amati G, et al (2012) Morphologic evidence of diffuse vascular damage in human and in the experimental model of ethylmalonic encephalopathy. J Inherit Metab ...
Effective AAV-mediated gene therapy in a mouse model of ...
Ethylmalonic encephalopathy (EE) is an invariably fatal disease, characterized by the accumulation of hydrogen sulfide (H 2 S), a highly toxic compound. ETHE1 , encoding sulfur dioxygenase (SDO), which takes part in the mitochondrial pathway that converts sulfide into harmless sulfate, is mutated in EE.