5.0 STUDY TREATMENT 
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(For additional information see Appendix V and VI) 
5.1 Justification of Dosages 
It is estimated that the ratio of total bone marrow cells to stem cells is 1 : 10 5 . With this 
assumption, then 10 4 to 10 5 stem cells will be exposed to the Gc vector from each 
patient. If the transduction efficiency is as low as 1 % , stem cell transduction should still 
be detectible in patients. Preclinical studies imply that the transduction frequency into 
progenitor cells may be as high as 50%. If stem cell transfer is affirmed, conditions for 
bone marrow or peripheral stem cell collection and transduction will be further 
optimized. 
5.2 Formulation. Packaging, and Storage 
More complete descriptions of these reagents are included in Appendix V. Brief 
descriptions are listed below: 
G-CSF 
Granulocyte colony stimulating factor is manufactured by Amgen. G-CSF is known to 
stimulate granulocytes and their precursors. It is used in this protocol to mobilize 
peripheral blood stem cells. 
SCF 
Stem cell factor is manufactured by Amgen. It is thought to act synergistically with 
other growth factors to promote growth of early hematopoietic cells. It is used in this 
protocol to increase the overall rate of gene transfer into the earliest progenitor cells to 
improve the efficiency of gene transfer. 
11^3 
Interleukin-3 is manufactured by Sandoz. It acts synergistically with a variety of growth 
factors to stimulate cell division of early marrow cells. It is used in this protocol because 
its use has been shown to increase the efficiency of gene transfer when retroviral vectors 
are used. 
IL-6 
Interleukin-6 is manufactured by Sandoz. Like the previous cytokines, it acts 
synergistically with other growth factors to stimulate hematopoietic cell division and 
increase the efficiency of gene transfer. 
Gc Retroviral Vector Supernatant 
Gc retroviral vector supernatant is manufactured by Genetic Therapy, Inc. It consist of 
the conditioned medium obtained from Gc producer cells. Gc producer cells have been 
engineered to produce retroviral vector particles with an amphotropic envelope so that 
the vector may be transferred into human cells. This vector is referred to as the LG 
vector in the published literature (20, 25). 
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Recombinant DNA Research, Volume 17 
