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Translational Research : the Journal of Laboratory and Clinical Medicine|January 29, 2023
AAV-based gene therapy prevents and halts the progression of dilated cardiomyopathy in a mouse model of phosphoglucomutase 1 deficiency (PGM1-CDG)Bijina Balakrishnan, Ruqaiah Altassan, Rohit Budhraja, et al.Molecular Therapy. Methods & Clinical Development|February 14, 2024
Whole-body galactose oxidation as a robust functional assay to assess the efficacy of gene-based therapies in a mouse model of GalactosemiaBijina Balakrishnan, Xinhua Yan, Marshall D McCue, et al.Journal of Translational Medicine|February 21, 2026
PGM1 deficiency is linked to sarcomeric and mitochondrial dysfunction in patient-derived iPSC-cardiomyocytesSilvia Radenkovic, Graeme Preston, Rohit Budhraja, et al.Journal of Medicinal Chemistry|September 7, 2021
Structure-Based Optimization of Small Molecule Human Galactokinase InhibitorsLi Liu, Manshu Tang, Rajan Pragani, et al.Pageof 2