patient-specific iPSCs

Amelioration of X-Linked Related Autophagy Failure in Danon Disease with DNA Methylation Inhibitor

Background—Danon disease is an X-linked disorder that leads to fatal cardiomyopathy, caused by a deficiency in lysosome-associated membrane protein-2 (LAMP2). In female patients, a later-onset and less severe clinical phenotype have been attributed …