been studied after treatment? Miat tests have been used 
and what is their sensitivity? 
3. Clinical procedures , including patient monitoring 
Describe the treatment that will be administered to patients and 
the diagnostic methods that will be used to monitor the success or 
failure of the treatment. If previous clinical studies using 
similar methods have been performed by yourself or others, indicate 
their relevance to the proposed study. 
a. Will cells (e.g., bone marrow cells) be removed frcm patients 
and treated in vitro in preparation for gene therapy? If so, 
what kinds of cells will be removed from the patients, how many, 
how often, and at what intervals? 
b. Will patients be treated to eliminate or reduce the number of 
cells containing malfunctioning genes (e.g., through radiation 
or chemotherapy) prior to gene therapy? 
c. What treated cells (or vector/DNA combination) will be given 
to patients in the attempt to administer gene therapy? How 
will the treated cells be administered? What volume of cells 
will be used? Will there be single or multiple treatments? 
If so, over what period of time? 
d. What are the clinical endpoints of the study? Are there 
objective and guantitative measurements to assess the natural 
history of the disease? Will such measurements be used in 
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