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• W2091608489 abstract "Abstract Many metabolic diseases are compelling candidates for gene therapy, and are the subject of vigorous pre‐clinical research. Successful phenotype correction in mouse models is now commonplace and research effort is increasingly being directed towards addressing the translational challenges inherent in human clinical trials. This paper places current efforts to develop gene therapy approaches to metabolic disease in historical context and describes contemporary research in the authors’ laboratory on urea cycle defects, particularly ornithine transcarbamylase deficiency, in a manner that is illustrative of the general state of the field." @default.
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• W2091608489 date "2012-03-09" @default.
• W2091608489 modified "2023-10-18" @default.
• W2091608489 title "Gene therapy for metabolic disorders: an overview with a focus on urea cycle disorders" @default.
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• W2091608489 doi "https://doi.org/10.1007/s10545-012-9467-0" @default.
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