an allergic reaction is theoretically possible; you (your child) 
would of course be treated for any reaction that occurred. It is 
possible that the cells could become infected with bacteria or 
fungus during the 3 day growth period in the laboratory. If 
there is any evidence for infection of the cells with unwanted 
agents, the cells will not be returned to you (your child), but 
there is a remote chance that you (your child) could acquire a 
bacterial or fungal infection from the reinfused cells. 
Although we do not believe that introduction of the mouse virus 
vector containing the Fanconi ' s anemia gene or the growth of your 
cells in tissue culture should be harmful, we can not be 
absolutely certain that no risks apply, especially to Fanconi ' s 
anemia patients. In Fanconi ' s anemia, there is a predisposition 
to leukemia and other cancers, and it is theoretically possible 
that these treatments might encourage the growth of leukemia 
cells . 
You (your child) will receive an infusion of cells three days 
after the end of your G-CSF treatment. 
OVERALL TREATMENT PLAN 
We will repeat this whole treatment procedure up to four times, 
with at least two months between each treatment. In other words, 
each patient will be treated with one week of G-CSF, cells will 
be removed by apheresis for gene therapy, and introduced back 
into the patient four days later. Two to three months later, 
this procedure will be repeated, up to a total of four 
treatments . 
FOLLOW-UP 
We will perform a number of blood tests to determine whether the 
normal Fanconi ' s anemia gene is present in your blood and bone 
marrow cells and whether it is affecting the way these cells grow 
in tissue culture. We will also perform tests to make sure the 
gene transfer procedure is safe. The amount of blood drawn will 
be approximately four tablespoons every month. We will also need 
to repeat bone marrow examinations at the time of each gene 
therapy. Because we need to ensure that the gene transfer 
procedure does not cause any harmful effects in the bone marrow 
we will exclude from the protocol patients who refuse these 
procedures . 
Since this study involves a very new approach to patients with 
Fanconi ' s anemia, it will be important to maintain contact with 
you (your child) for the rest of your life so that we can monitor 
your overall progress and monitor for any possible long term side 
effects. If the Fanconi ' s gene is successfully transferred into 
stem cells, your body might contain the cells carrying the 
transferred gene for life. We would continue to see you (your 
child) in our clinic yearly for at least five years. In the 
event of your death, we would request an autopsy for the purpose 
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