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, Willisa Liou, Arielle Lupo, Sarah L. Bryant, Anastasiya Mankouski, Silvia Radenkovic, Graeme Preston, Akhilesh Pandey, Sihem Boudina, Tamás Kozicz, Éva Morava, Kent Lai
Topics & Concepts
BiologyDilated cardiomyopathyEjection fractionCardiomyopathyPhosphoglucomutaseInternal medicineEndocrinologyHeart failurePathologyMedicineBiochemistryEnzymeDigestive system and related healthInfant Nutrition and HealthPancreatic function and diabetes