Attachment VI - Pago 10 
12 
a. Will cells (e.g. , hone marrow cells) be removed from 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? Vhat volume of cells 
will be used? Will there be single or multiple treatments? 
If so, over what period of time? 
d. Vhat are the clinical endpoints of the study? How will patients 
be monitored to assess specific effects of the treatment on the 
disease? What is the sensitivity of the analyses? How frequently 
will follcw-up studies be done? How long will patient follcw-up 
continue? 
e. What are the major potential beneficial and adverse effects of 
treatment that you anticipate? Vhat measures will be taken in 
an attempt to control or reverse these adverse effects if they 
occur? Compare the probability and magnitude of potential 
adverse effects on patients with the probability and magnitude 
of deleterious consequences from the disease if gene therapy 
is not performed. 
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