Julie Saba

AAV-SPL, a One-Time Gene Therapy for Rare and Common Forms of Fibrotic Kidney Disease

National Institute of Diabetes and Digestive and Kidney Diseases · R42

Contact PI · $352.2K · Sphinxion Therapeutics, Inc

Characterizing the Natural History of Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS): A Fundamental Step in the Development of a Targeted Cure for This Novel Atypical Sphingolipidosis

National Institute of Child Health and Human Development · R01

Contact PI · $2.3M · University of California, San Francisco

Adeno-Associated Virus-Mediated SGPL1 Gene Therapy as a First-in-Class Treatment for Idiopathic Pulmonary Fibrosis That Acts by Reducing Sphingosine-1-Phosphate and Downstream Profibrotic Signaling

National Heart, Lung, and Blood Institute · R33

Contact PI · $574K · University of California, San Francisco

Proof-of-Concept for the Use of Enzyme Replacement Therapy as a Lifesaving Treatment for Sphingosine Phosphate Lyase Insufficiency Syndrome

National Center for Advancing Translational Sciences · R21

Contact PI · $463.2K · University of California, San Francisco

SPL Insufficiency Syndrome (SPLIS)/NPHS14: a SPLIS Observational Study and Patient Registry (International)

Principal investigator · Recruiting

University of California, San Francisco

Natural History of Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)

Principal investigator · Recruiting

University of California, San Francisco

Role of Biomarkers in Disease Progression in Samples From Patients With Acute Myeloid Leukemia

Principal investigator · Completed

Children's Oncology Group