| Quest – Discovery Stage Research Projects | University of California, Los Angeles | Gene Therapy for SLC6A8 Creatine Transporter Disorder |
$2,296,920 |
| Therapeutic Translational Research Projects | University of California, Los Angeles | Development of a Gene Therapy for Treatment of Guanidinoacetate Methyltransferase Deficiency-Translating In Vivo Proof of Concept to Support a Pre-IND |
$5,145,825 |
| Therapeutic Translational Research Projects | University of California, Los Angeles | Development of a Gene Therapy for the Treatment of Arginase Deficiency – Translating from Proof of Concept to Support Pre-IND Meeting |
$5,266,504 |
| Early Translational IV | University of California, Los Angeles | Gene therapy-corrected autologous hepatocyte-like cells from induced pluripotent stem cells for the treatment of pediatric single enzyme disorders |
$1,801,629 |
