Sickle Cell Disease

Gene Transfer for Patients With Sickle Cell Disease

Gene Transfer for Patients With Sickle Cell Disease Using a Gamma Globin Lentivirus Vector: An Open Label Phase I/II Pilot Study

Phase 1/2


Researchers at Cincinnati Children’s Hospital Medical Center are recruiting patients with Sickle Cell Disease / Sickle Cell Anemia for a genetic transfer investigational treatment. The goal of the treatment is to provide sickle cell patients with the gene needed to create normal red blood cells (RBCs).

This investigatory treatment requires surgical recovery of the patient’s bone marrow, which will then be gene-edited using a harmless virus to transfer the correct gene into the patient’s bone marrow. The patient will undergo chemotherapy prior to the treatment, then the gene-edited bone marrow will be transfused back into the patient. Follow up will be approximately 15 years after this important study.


  • Be 18 to 35 years of age
  • Able to undergo general or regional anesthesia
  • Willing to reside within 120 miles of Cincinnati Children’s Hospital for 3 to 6 months
  • Not be HIV-positive
  • Not be pregnant or breastfeeding
  • Not have alpha thalassemia


  1. Pre-screening at the study site to confirm eligibility of the patient to participate.
  2. A surgical bone marrow harvest to collect blood making cells.
  3. The gene-editing phase of the study will not require patient involvement.
  4. Chemotherapy treatment will reduce the patient’s bone marrow.
  5. One-time Infusion of the investigational drug, with close observation period.
  6. Patients will be followed for ~ 15 years.


Cincinnati Children’s Hospital Medical Center in Cincinnati, OH, is recruiting participants for this trial. Map.

Primary contact: Amy Shova | [email protected] | 513-636-6770


Children’s Hospital Medical Center, Cincinnati & Doris Duke Charitable Foundation

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