Primary LAMP-2 deficiency causes X-linked vacuolar cardiomyopathy and myopathy (Danon disease)
Microdeletions in the 5′ region of LAMP2 gene associated with Danon disease
Human iPSC model of Danon disease reveals LAMP2 haploinsufficiency and rescue by gene therapy
A novel LAMP2 mutation associated with severe cardiac hypertrophy and microvascular remodeling
Danon Disease: Entire LAMP2 Gene Deletion with Unusual Clinical Presentation
Danon disease: a phenotypic expression of LAMP-2 deficiency
Systemic AAV9.LAMP2B injection reverses multiorgan dysfunction in a murine model of Danon disease
Phase 1 Study of AAV9.LAMP2B Gene Therapy in Danon Disease
Lysosomal membrane protein Lamp-2 is required for autophagy in the mouse heart