Grant Award Details
Identify a therapeutic candidate for Danon Disease comprising autologous, HSPCs transduced with lentivirus containing the human LAMP2 cDNA, and to demonstrate disease-modifying activity in LAMP2 KO mice with analogous murine product
Grant Application Details
- Genetically Modified Hematopoietic Stem Cells for the Treatment of Danon Disease
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.
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.
Major Proposed Activities
- Generation of ex vivo Genetically Modified Human HSPC Product (Month 1-6)
- Functional Characterization of ex vivo Genetically Modified Human HSPC Product
- Generation of Analogous Murine Product
- In vivo Efficacy Evaluation of Analogous Murine Product in the Mouse Model of Danon Disease
- Elucidate Purported Mechanism of Action
Danon disease is a fatal disease without cure, therefore the cellular treatment we plan to develop could directly benefit the citizens of
California. Our findings may assist in the development of new treatments for other cardiac diseases. Thus the work also
has the potential to help Californians who suffer from similar cardiac conditions. This project utilizes CA scientists and laboratories. With success, it will generate additional research and employment opportunities for CA citizens.