Effective AAV-mediated gene therapy in a mouse model of ethylmalonic encephalopathy

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...

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Main Authors: Di Meo, Ivano, Auricchio, Alberto, Lamperti, Costanza, Burlina, Alberto, Viscomi, Carlo, Zeviani, Massimo
Formato: Artigo
Idioma:Inglês
Publicado: WILEY-VCH Verlag 2012
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Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC3491831/
https://ncbi.nlm.nih.gov/pubmed/22903887
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/emmm.201201433
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