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Correction of Methylmalonic Aciduria In Vivo Using a Codon-Optimized Lentiviral Vector

Methylmalonic aciduria is a rare disorder of organic acid metabolism with limited therapeutic options, resulting in high morbidity and mortality. Positive results from combined liver/kidney transplantation suggest, however, that metabolic sink therapy may be efficacious. Gene therapy offers a more a...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Hauptverfasser: Wong, Edward S.Y., McIntyre, Chantelle, Peters, Heidi L., Ranieri, Enzo, Anson, Donald S., Fletcher, Janice M.
Format: Artigo
Sprache:Inglês
Veröffentlicht: Mary Ann Liebert, Inc. 2014
Schlagworte:
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4064729/
https://ncbi.nlm.nih.gov/pubmed/24568291
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2013.111
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