Disease Focus: Danon Disease

---

Genetically Modified Hematopoietic Stem Cells for the Treatment of Danon Disease

Research Objective We propose to discover a novel, genetically modified hematopoietic stem cell based treatment for Danon disease, a rare lysosomal storage disease that affects the heart. Impact As the only existing treatment for Danon disease is cardiac transplant, this therapy would significantly meet an unmet need. It also may help many other similar diseases. […]