Synergistic Deoxynucleoside and Gene Therapies for Thymidine Kinase 2 Deficiency.

Carlos Lopez-GomezMaria J Sanchez-QuinteroEung Jeon LeeGulio KleinerSaba TadesseJun XieHasan Orhan AkmanGuangping GaoMichio Hirano

Published in: Annals of neurology (2021)

Our data indicate that AAV-TK2 gene therapy as well as combination deoxynucleoside and gene therapies is more effective in Tk2KI mice than pharmacological alone. Thus, combination of gene therapy with substrate enhancement is a promising therapeutic approach for TK2 deficiency and potentially other metabolic disorders. ANN NEUROL 2021;90:640-652.

Keyphrases

gene therapy
copy number
genome wide
genome wide identification
replacement therapy
electronic health record
big data
high fat diet induced
type diabetes
cancer therapy
gene expression
neural network
protein kinase
drug delivery
genome wide analysis
skeletal muscle