New hope for sickle cell cure: High-Dose drug trial aims to unlock gene therapy
NCT ID NCT07341022
Summary
This study is testing if a high dose of a drug called plerixafor can safely and effectively collect enough stem cells from people with sickle cell disease. These collected cells are a necessary first step for future gene therapy treatments, which aim to correct the disease at its genetic root. The trial will enroll 12 young adult patients in Brazil to see if this approach works and is safe for this population.
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