Therapeutic Translational Research Projects | University of California, Davis | Overexpression of HexA/HexB by lentivector expression in blood cells to treat Tay-Sachs and Sandhoff disease |
$883,174 |
Late Stage Preclinical Projects | University of California, Davis | Expression of Ube3a by the hematopoietic system for the treatment of Angelman syndrome |
$4,487,656 |
Late Stage Preclinical Projects | University of California, Davis | Hematopoetic stem cell gene therapy for the treatment of Tay-Sachs disease |
$4,048,253 |