Synergistic Deoxynucleoside and Gene Therapies for Thymidine Kinase 2 Deficiency
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DOI: 10.1002/ana.26185
Abstract: Autosomal recessive human thymidine kinase 2 (TK2) mutations cause TK2 deficiency, which typically manifests as a progressive and fatal mitochondrial myopathy in infants and children. Treatment with pyrimidine deoxynucleosides deoxycytidine and thymidine ameliorates mitochondrial defects… read more here.
Keywords: synergistic deoxynucleoside; therapy; gene; deficiency ... See more keywords