that harm Fanconi ' s anemia cells, and a more normal appearance of 
the cells' chromosomes. Because the cells containing the normal 
Fanconi ' s anemia type C gene grow better, these cells may have a 
competitive advantage compared to unaltered Fanconi ' s anemia type 
C cells. 
The purpose of this research protocol is to test whether we can 
safely introduce the normal Fanconi ' s anemia type C gene into 
CD34 cells of patients with this disease. As described below in 
more detail, CD34 cells are primitive blood cells that include 
stem cells. Stem cells are the cells in the bone marrow and 
blood that give rise to the white cells, platelets and red cells. 
We will treat blood cells from patients with Fanconi ' s anemia 
type C using the viral carrier containing the Fanconi ' s anemia 
gene in a test tube and then return these cells to patients. If 
the cells are genetically altered, we expect to be able to detect 
the normal gene in blood and bone marrow cells afterwards. We 
hope that cells that contain the normal Fanconi ' s anemia gene 
will grow well in the bone marrow, and that we will be able to 
detect normal cells in special tissue culture studies. If we are 
successful, we should also be able to correct the chromosome 
abnormality. It may even be possible to increase blood counts in 
patients with Fanconi ' s anemia using this procedure. However, it 
should be clear that the major purpose of this protocol is to 
test the safety of this technique and to determine whether we can 
transfer the Fanconi ' s anemia gene successfully. We do not 
expect that this protocol will decrease the risk of cancer in the 
other organs of the body. 
Potential benefits : We do not know if there will be any direct 
benefit to you (your child) from participating in this study. 
The major goal of this study is to determine if the Fanconi ' s 
anemia gene can be transferred safely to patients with type C 
disease. However, there may be future benefits to you (your 
child) and to other patients with Fanconi ' s anemia. It is 
possible that the results of this study will lead to a new form 
of experimental therapy for Fanconi ' s anemia. It is also possible 
that more prolonged courses of gene therapy would be clinically 
beneficial. You (your child) would be considered for a such a 
future protocol . 
In order to obtain sufficient CD34 cells from patients with 
Fanconi ' s anemia, we will treat each patient with a hematopoietic 
growth factor called granulocyte -colony stimulating factor or G- 
CSF. G-CSF will be given for one week, at the end of which time 
blood cells will be removed by a process called apheresis for 
treatment with the Fanconi ' s anemia type C gene vector. 
PRESTUDY TESTING 
Prior to being entered into this study, you (your child) will 
undergo blood tests, bone marrow aspiration and biopsy, as well 
as a full history and physical examination to determine the 
status of your Fanconi 's anemia. You (your child) will have 
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