Article

Gene therapy for inborn errors of metabolism

2013-01-01

Abstract excerpt

Inborn errors of liver metabolism are frequent causes of morbidity and mortality especially in children. For several of these diseases, treatment approaches depend on manipulation of the affected metabolic pathway by diet, drugs, vitamin cofactors, enzyme induction, end-product replacement, and alternative pathway activation. Unfortunately, these approaches often remain unsatisfactory especially in the face of ill...

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Literature Corpus work: 8a19f241-d99f-5ee2-baf2-47cefc66010d
DOI: 10.21954/ou.ro.0000f049

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