Clinical trial
Stem Cell Gene Therapy for Cystinosis
Abstract
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, including product manufactured with and without the transduction enhancer LentiBOOST \[CTNS-RD-04-LB\])
Topics & conditions
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Keywords
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