GTI0101 (04/08/93) 
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SCHEMATIC 
Peripheral Blood Stem Cell Harvest 
Patients with Gaucher Disease 
G-CSF 10 ug/kg/day 
(Day 1-7) 
Peripheral Blood Stem Cell Harvest 
Filtered and processed to recover CD34-enriched Cells 
(Days 5, 6, and 7) 
Transduced with GC-gene containing retroviral vector(s) 
(Days 5-10) 
Transduced cells reinfused into patient 
(Day 8 - 10) 
Patient Discharged 
(Day 11) 
Patient Follow-up 
(end of Cycle 1) 
Repeat up to 3 more cycles 
[792] 
Recombinant DNA Research, Volume 17 
