minimizing the problems associated with Gaucher Disease if an 
appropriate donor is available. However, the majority of 
patients will not have a donor. In addition there are substantial 
health risks associated with a bone marrow transplant. Since a 
bone marrow transplant is often not possible, alternative 
treatments have been tested. The best available treatment for 
Gaucher Disease is known as enzyme replacement therapy. Enzyme 
replacement therapy with "Ceredase" , the commercially available 
product, is effective and safe in most patients, and you may be 
receiving this therapy at the present time. However, enzyme 
replacement therapy must continue for the entire life of a 
Gaucher disease patient, is very expensive, costing about 
$100,000 to $200,000 per year, and may not be available to many 
patients with Gaucher disease. 
The purpose of this study is to investigate an alternative 
form of therapy for Gaucher Disease called "gene therapy". We 
seek to determine if the normal gene responsible for producing 
glucocerebrosidase can be placed into the blood or bone marrow 
cells of patients with Gaucher Disease. Certain cells in the 
bone marrow or blood, called "stem cells," make all of the blood 
cells in the body, such as red blood cells, white blood cells and 
platelets. One type of white blood cell made by these stem 
cells, called a "macrophage," is the cell that builds up the 
abnormal waste products in Gaucher disease. If we could insert 
the normal gene for the missing GC enzyme into the stem cells 
which make macrophages, then possibly these macrophages would no 
longer build up the toxic waste products, and some of the 
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