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Lysosomal Storage Diseases clinical trials at UCSD

1 in progress, 0 open to eligible people

Showing trials for
  • Stem Cell Gene Therapy for Cystinosis

    Sorry, in progress, not accepting new patients

    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 or CTNS-RD-04-LB, where the suffix "-LB" stands for LentiBOOST)

    La Jolla, California

Our lead scientists for Lysosomal Storage Diseases research studies include .

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