Gene Transfer for Patients With Sickle Cell Disease

The purpose is also to determine if this kind of gene transfer works in getting the body to
make red blood cells that don't sickle in patients with sickle cell disease.

Inclusion Criteria:

- Confirmed sickle cell disease that is severe

- Failed hydroxyurea therapy, were unable to tolerate hydroxyurea therapy, or, if 18
years of age or older, have actively made the choice to not take the recommended daily
hydroxyurea. Patients who are unable to take hydroxyurea due to financial or safety
monitoring constraints are eligible.

- Lansky/Karnovsky performance score≥ 60

- Ability to undergo general or regional(spinal or epidural) anesthesia

- Adequate kidney, liver and heart function as determined by clinical tests

- Willing to reside within a 120 mile radius of Cincinnati Children's Hospital Medical
Center for a period of 3-6 months

Exclusion Criteria:

- Active malignant disease

- Sero-positive for HIV

- Taken an experimental drug in past 30 days

- Abnormal pulmonary function tests that show severe obstruction, restriction or
diffusion defects

- Patients who are on chronic transfusions for:

- clinical stroke

- Pregnant or breast-feeding

- Alpha thalassemia

- Cirrhosis of liver, bridging hepatic fibrosis, or active hepatitis

- Patients with matched sibling donors who have not declined the option of a
hematopoietic stem cell transplant

- Confirmed pulmonary hypertension

- Any condition which in the opinion of the investigator makes participation ill advised