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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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Detalles Bibliográficos
Autores principales:	Di Meo, Ivano, Auricchio, Alberto, Lamperti, Costanza, Burlina, Alberto, Viscomi, Carlo, Zeviani, Massimo
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	WILEY-VCH Verlag 2012
Materias:	Report
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3491831/ https://www.ncbi.nlm.nih.gov/pubmed/22903887 http://dx.doi.org/10.1002/emmm.201201433

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