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Note for Peripheral Blood Stem Cell (PBSC) Harvest: 
Days PI - P7 : Begin administration of G-CSF to patient 
Days P5. P6. and P7 : PBSC harvested 
Days P5 - P10 : PBSC transduction ex vivo with GC vector 
Days P8. P9. and P10 : Transduced cells reinfused 
Day PI 1 : Patient discharge 
4.21 Objective signs and symptoms : Vital signs (blood pressure, pulse, temperature 
and respirations) will be done Study Days Ml, M2, M4, M5 and PI through PI 1. 
Weight will be done day of discharge (M5, PI 1) 
4.22 Physical Examination : Target physical exam will be repeated day of discharge 
(M5, Pll). 
4.23 Kamofskv Performance Status (see Appendix IV) will be done day of discharge 
(M5, Pll). 
4.24 Urinalysis : Routine urinalysis, including creatinine and creatinine clearance, will 
be done Day Ml, M2, M4, and Pl-10 and day of discharge (M5, Pll). 
4.25 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 
will be done Study Days Ml, M2, M4,and P1-P10 and day of discharge (M5, 
Pll). 
4.26 Hematology : Complete blood count (CBC), differential, platelets, and 
coagulation tests should include PT (Prothrombin Time), PTT (Partial 
Thromboplastin Time), and fibrinogen will be done Study Days Ml, M2, M4 
and P1-P10 and day of discharge (M5, Pll). 
4.27 Pregnancy Test : Females will have a serum pregnancy test repeated monthly 
during the first year. 
4.28 Liver-Spleen Size by MRI or CAT : Optional, if clinically indicated. 
4.29 Glucocerebrosidase enzyme level determination : Obtain PBMC on day of 
discharge. 
4.210 MoMLV Antibody Analysis : Sample obtained day of discharge (Day M5, Pll). 
4.211 Freezing Specimens : Additional serum, plasma, and cells should be separated 
and frozen day of discharge (Day M5, Pll). 
4.212 DNA/RNA Analysis : Circulating mononuclear cells will be analyzed using PCR- 
related techniques for the presence of vector gene sequences day of discharge. 
Recombinant DNA Research, Volume 17 
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