Associated Papers
Navigating molecular therapies: The emerging role of mRNA in treating inherited metabolic diseases.
Castro-Alpízar JA et al.
Molecular Genetics and Metabolism, 2026
Rapid targeted analysis of the genome: Rapid genomic sequencing in critically ill infants.
Wild KT et al.
Genetics in Medicine Open, 2026
41584744 (PubMed )
Supplement: mmc4.xlsx › Sheet1
Improved specificity and efficiency of in vivo adenine base editing therapies with hybrid guide RNAs.
Whittaker MN et al.
Nature Biomedical Engineering, 2025
Amino Acid Metabolism in Liver Mitochondria: From Homeostasis to Disease.
Erdal R et al.
Metabolites, 2025
Personalized Gene Editing to Treat an Inborn Error of Metabolism.
Gropman AL et al.
The New England Journal of Medicine, 2025
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease.
Musunuru K et al.
The New England Journal of Medicine, 2025
NAGS, CPS1, and SLC25A13 (Citrin) at the Crossroads of Arginine and Pyrimidines Metabolism in Tumor Cells.
Owusu-Ansah M et al.
International Journal of Molecular Sciences, 2023
37047726 (PubMed )
Supplement: ijms-24-06754-s001.zip › ijms-2241907-supplementary › Supplementary_Tables_Figures › Supplementary_Table_S10.xlsx › CPS1_Patients
Variants associated with urea cycle disorders in Japanese patients: Nationwide study and literature review.
Kido J et al.
American Journal of Medical Genetics. Part A, 2021
Hyperammonemia in urea cycle disorders: A toxic metabolite for the brain.
Kido J et al.
Pediatrics International : Official Journal of the Japan Pediatric Society, 0
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