GTI0101 (04/08/93) 
Page 1 9 of 35 
4.17 Serum Chemistries : include uric acid, calcium, phosphorus, magnesium, amylase, 
transaminases (SGOT, SGPT), alkaline phosphatase, LDH, total bilirubin, 
triglycerides, BUN, creatinine, albumin, total protein, electrolytes, and glucose 
(Screening and repeat Day 0) 
4.18 Hematology : Complete blood count (CBC), differential, platelets, ESR, and 
coagulation tests should include PT (Prothrombin Time), PTT (Partial 
Thromboplastin Time), and fibrinogen (Screening and repeat Day 0). 
4.19 Pregnancy Test : Serum pregnancy test must be negative within 7 days prior to 
Day 0, or between study days -7 and 0. 
4. 1 10 HIV Negative Antibody Test : ELISA (Screening) 
4.111 HBsAG : Hepatitis B surface antigen determination (Screening) 
4.112 Liver-Spleen Size by MRI or CAT : Optional: To be done if clinically indicated. 
4.113 Resting transcutaneous oxygen saturation : Must be > 90% on room air 
4.114 Glucocerebrosidase enzyme level : collect PBMCs for this determination 
(Screening) 
4.115 MoMLV Antibody Analysis : (Day 0) 
4.116 Freezing Specimens : Additional serum, plasma, and blood cells should be 
separated and frozen at baseline (Day 0). 
4.117 DNA/RNA Analysis : Circulating mononuclear cells will be analyzed using PCR- 
related techniques for the presence of vector gene sequences (Day 0, baseline). 
4.118 Marrow MRI Analysis : Analysis of marrow involvement will performed using 
T1 weighted magnetic resonance imaging techniques if clinically warranted. 
Evaluations During Treatment : 
Evaluations are to be done prior to that days procedures or treatments. 
Note for Marrow Harvest: 
Day Ml : Marrow harvest 
Dav M2 : Patient discharged if clinically indicated 
Days Ml. M2, and M3 : CD34+ cell transduction ex vivo with Gc 
Day M4 : Transduced marrow reinfusion (in- or out-patient) 
Day M5 : Patient discharge 
Recombinant DNA Research, Volume 17 
