the transduction frequency into progenitor cells may be 
as high as 50% (20). 
3 . 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. SCF acts 
synergistically with other growth factors to promote 
growth of early hematopoietic cells. It is used in 
this protocol to increase the efficiency of gene 
transfer into the earliest progenitor cells. 
IL-3 
Interleukin- 3 is manufactured by Sandoz. IL-3 acts 
synergistically with a variety of growth factors to 
stimulate cell division of early marrow cells, and it 
is used in this protocol because its use has been shown 
to increase the efficiency of retroviral gene transfer. 
IL-6 
Interleukin- 6 is manufactured by Sandoz. IL-6 acts 
synergistically with other growth factors to stimulate 
hematopoietic cell division and, in general, increases 
the efficiency of gene transfer. 
FACC Retroviral Vector Supernatant 
FACC retroviral supernatant is manufactured by Genetic 
Therapy, Inc. It consists of the conditioned medium 
obtained from producer cells. These producer cells 
have been engineered to produce retroviral vector 
particles with an amphotropic envelope so that the 
vector may be transferred into human cells. 
3 . 3 Treatment Procedures 
(Refer to section 6.0 for dose modification and 
toxicity management) 
3.31 Harvest of Hematopoietic Stem Cells 
1 . Peripheral Blood Stem Cell Harvest : 
Peripheral blood stem cells will be harvested 
after mobilization with the growth factor G-CSF. 
Study Days 0-6 : G-CSF will be given at a dose of 
lO/xg/kg/day subcutaneously for a period of 7 
consecutive days. The drug will be given as a 
single daily injection, and the patient may 
receive acetominophen or other non- aspirin 
analgesics for any possible associated fever or 
bone pain. 
Study Days 5-7 : On days 5, 6, and 7, the patient 
will undergo apheresis procedures for the 
collection of peripheral blood mononuclear cell 
fractions with an expected maximum yield of 1 x 
10® cells per kilogram body weight. 
3.32 Separation of CD34+ Population of Hematopoietic Cells : 
All the cells from the peripheral blood stem cell 
Recombinant DNA Research, Volume 19 
