Gene Therapy for SPLIS: Advancing a Cure for Rare Kidney Disease @UniversityHospitals
Gene Therapy for SPLIS: Advancing a Cure for Rare Kidney Disease @UniversityHospitals
Uploaded April 2026 | Updated September 2026, 1 week ago
Julie Saba, MD, University of California, San Francisco, 2024 Harrington Scholar-Innovator, is developing a gene therapy to treat sphingosine-1-phosphate lyase insufficiency syndrome (SPLIS), a fatal childhood kidney disorder, with the goal of offering a life-saving, curative option for affected patients.

Harrington Discovery Institute at University Hospitals in Cleveland, Ohio aims to accelerate promising discoveries into medicines for unmet needs. Learn more: HarringtonDiscovery.org.
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Gene Therapy for SPLIS: Advancing a Cure for Rare Kidney Disease

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