Deoxynucleoside therapy for diseases caused by unbalanced nucleotide pools including mitochondrial DNA depletion syndromes

The invention relates generally to a pharmacological therapy for human genetic diseases, specifically those characterized by unbalance nucleotide pools, more specifically mitochondrial DNA depletion syndromes, and more specifically, thymidine kinase 2 (TK2) deficiency. The pharmacological therapy in...

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Hauptverfasser:	The Trustees of Columbia University in the City of New York, Fundació Hospital Universitari Vall d'Hebron-Institut de Recerca
Format:	Patent
Sprache:	eng
Online-Zugang:	Volltext bestellen
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