Neonatal gene therapy achieves sustained disease rescue of maple syrup urine disease in mice

Maple syrup urine disease (MSUD) is a rare recessively inherited metabolic disorder causing accumulation of branched chain amino acids leading to neonatal death, if untreated. Treatment for MSUD represents an unmet need because the current treatment with life-long low-protein diet is challenging to...

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Detalles Bibliográficos
Autores principales: Pontoizeau, Clément, Simon-Sola, Marcelo, Gaborit, Clovis, Nguyen, Vincent, Rotaru, Irina, Tual, Nolan, Colella, Pasqualina, Girard, Muriel, Biferi, Maria-Grazia, Arnoux, Jean-Baptiste, Rötig, Agnès, Ottolenghi, Chris, de Lonlay, Pascale, Mingozzi, Federico, Cavazzana, Marina, Schiff, Manuel
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9174284/
https://www.ncbi.nlm.nih.gov/pubmed/35672312
http://dx.doi.org/10.1038/s41467-022-30880-w

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