Neonatal gene therapy achieves sustained disease rescue of maple syrup urine disease in mice
Neonatal gene therapy achieves sustained disease rescue of maple syrup urine disease in mice
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Author / Creator
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 and Schiff, Manuel
Publisher
London: Nature Publishing Group UK
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Language
English
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Publisher
London: Nature Publishing Group UK
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Scope and Contents
Contents
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 maintain, and despite treatment the risk of acute...
Alternative Titles
Full title
Neonatal gene therapy achieves sustained disease rescue of maple syrup urine disease in mice
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TN_cdi_doaj_primary_oai_doaj_org_article_bccd1d42d05b472e924ee795b21fc179
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_bccd1d42d05b472e924ee795b21fc179
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ISSN
2041-1723
E-ISSN
2041-1723
DOI
10.1038/s41467-022-30880-w
