problems associated with Gaucher disease could be eliminated. 
The placement of the normal gene will be done by removing 
some bone marrow from the patient with Gaucher Disease, and then 
treating the bone marrow cells in the laboratory to place the 
glucocerebrosidase gene into the cells. The treated cells will 
then be placed back in the patient's body. We will then perform 
a number of tests to determine whether the new gene is present, 
and whether or not it is working normally. It is important to 
realize at this stage that this experimental procedure is only 
designed to test the safety of these new procedures and that it 
is not expected that the procedure will result in any direct 
medical benefit to you at this time. 
A number of private companies are supplying materials for 
the gene transfer part of the study. Genetic Therapy 
Incorporated (Gaithersburg, MD) is a company that makes the virus 
that delivers the normal GC gene into cells. Amgen Corporation 
(Thousand Oaks, CA) and Sandoz Corp (East Hanover, NJ) make 
growth factors that will be used to treat the bone marrow cells 
in the laboratory. CellPro Corp. (Seattle WA) makes a product 
that will be used to purify cells from the bone marrow. Each of 
these products is under review by the Food and Drug 
Administration (FDA) to determine if they are safe and effective. 
Because these four companies are supplying these reagents, they 
reserve the right to review the records of the treatment. They 
will maintain your confidentiality, as described on page 8. 
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Recombinant DNA Research, Volume 17 
