effects during and after the infusion. 
Following this procedure, you will be followed as an 
outpatient approximately every month for at least one year. We 
will perform a number of blood tests to determine whether the 
gene is present in your blood and bone marrow cells, and whether 
the gene is working normally. The amount of blood drawn will be 
approximately two-four tablespoons every month. We will request 
to perform a bone marrow aspiration test from one-to-six times 
over a two year period. You may refuse to undergo the bone 
marrow aspirations, if you wish, without being excluded from any 
other parts of the study. Other X-ray tests and scans may be 
performed as necessary for your routine clinical care. 
Since this study involves a very new approach to patients 
with Gaucher Disease, it would be important for us to maintain 
contact with you for at least five years, so that we can monitor 
your overall progress and determine any possible long term side 
effects. We request that, if you agree to participate in this 
study, you do not enter any other gene therapy studies for the 
next two years, so that we may monitor the results of this study. 
In the event of your death, we would request an autopsy for the 
purpose of determining whether the gene has been transferred into 
important organs, such as the liver or spleen. 
It is important to note that participation in this study 
will in no way alter any of the standard care you may be 
receiving. If you are currently receiving enzyme replacement 
therapy, it will continue. There will be no charge to you or 
your insurance carrier for any of the tests, procedures, or 
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Recombinant DNA Research, Volume 17 
