Prodrugs of Deoxynucleosides for Treatment of Mitochondrial Diseases Caused by Unbalanced Nucleotide Pools

Fecha de publicación: 05/01/2023
Fuente: WIPO Rice
Deoxynucleotide prodrugs and methods of their use for treatment of diseases characterized by unbalanced nucleotide pools such as MPV17 and deoxyguanosine kinase deficiency are provided herein.