Study Summary
This Phase I clinical trial will assess the safety and initial evidence for efficacy of an autologous transplant of lentiviral vector modified peripheral blood for adults with severe sickle cell disease.
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βAS3-FB vector transduced peripheral blood CD34+ cellsBIOLOGICAL
CD34+ from the peripheral blood of patients with sickle cell disease (SCD) are transduced ex-vivo with the Lenti/βAS3-FB lentiviral vector. The transduced cells are then infused into the patient.
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| University of California, Los Angeles (UCLA) | Los Angeles | California | United States |