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Lysosomal Storage Diseases clinical trials at UCSD
1 research study open to eligible people

  • Stem Cell Gene Therapy for Cystinosis

    open to eligible people ages 18 years and up

    This study is a Phase 1/2 clinical trial that will assess the safety and efficacy of enriched gene-corrected hematopoietic stem cells isolated from patients affected with cystinosis. (Investigational Product: CTNS-RD-04)

    La Jolla, California

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