Ethylmalonic encephalopathy and liver transplantation: long-term outcome of the first treated patient

BACKGROUND: Ethylmalonic encephalopathy (EE) is a severe intoxication-type metabolic disorder with multisystem clinical features and leading to early death. In 2014, based on the promising results obtained by liver-targeted gene therapy in Ethe1(−/−) mouse model, we successfully attempted liver tran...

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Detalles Bibliográficos
Autores principales: Olivieri, Giorgia, Martinelli, Diego, Longo, Daniela, Grimaldi, Chiara, Liccardo, Daniela, Di Meo, Ivano, Pietrobattista, Andrea, Sidorina, Anna, Semeraro, Michela, Dionisi-Vici, Carlo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2021
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8136189/
https://www.ncbi.nlm.nih.gov/pubmed/34011365
http://dx.doi.org/10.1186/s13023-021-01867-5

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