monitored in the hospital overnight. If no adverse reactions are observed, they will be 
discharged the following day for subsequent outpatient follow-up by the referring 
physician. 
Frequency of infusion of transduced CD34 + peripheral blood cells 
Peripheral blood collection, transduction and infusion will be repeated two more times at 
two month intervals for a total of three infusions. 
Evaluation 
A. Pretransplant Evaluation 
The following clinical and laboratory evaluations will be performed for all patients within 
30 days before start of treatment by the referring physician (screening). These screening 
evaluations must be reviewed prior to initiation of treatment. Certain evaluations will be 
repeated on the day of infusion of transduced CD34 peripheral blood cells (Day of 
transplant) prior to infusion. 
1. Complete history and physical examination (screening and repeat physical exam 
on Day of transplant). 
2. Karnofsky Performance Status (screening). 
3. Chest x-ray (PA and LAT) (screening). 
4. Electrocardiogram (screening). 
5. Urinalysis (screening and repeat on Day of transplant). 
6. Serum Chemistries: uric acid, calcium, phosphorus, magnesium, amylase, 
transaminases (SGOT, SGPT), alkaline phosphatase, LDH, total bilirubin, 
triglycerides, BUN, creatinine, albumin, total protein, electrolytes, glucose, non- 
tartrate inhibitable acid phosphatase and angiotensin converting enzyme 
(screening and repeat Day of transplant). 
7. Hematology: Complete blood count (CBC), differential, platelets, PT 
(Prothrombin Time), PTT (Partial Thromboplastin Time), and fibrinogen 
(screening, during G-CSF treatment, and on Day of transplant). 
8. Pregnancy Test: Serum pregnancy test must be negative within 7 days prior to 
Day of transplant. 
9. HIV Negative Antibody Test: ELISA (screening). 
10. HBsAG: Hepatitis B surface antigen determination (Screening). 
11. Liver-Spleen Size by MRI or CAT: In patients with clinical hepatosplenomegaly. 
12. Resting transcutaneous oxygen saturation: Must be > 90% on room air. 
13. Glucocerebrosidase enzyme level: collect PBMCs for this determination 
(screening). 
14. Freezing Specimens: Serum, plasma, and blood cells will be separated and frozen 
as baseline (Day of transplant). 
15. DNA/RNA Analysis: Circulating mononuclear cells will be analyzed using PCR- 
related techniques for the presence of vector gene sequences (Day of transplant). 
16. Marrow MRI Analysis: In patients with bone involvement. 
17. X-ray of bones affected by disease. 
18. Glucocerebrosidase gene mutation. 
(Send samples for 13-15 and 18 to Dr. Schuening’s lab.) 
Recombinant DNA Research, Volume 18 
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