Attachment II - Page 14 
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b. Will patients be treated to eliminate or reduce the number of 
cells containing rral functioning genes (e.g. , through radiation 
or chemotherapy) prior to gene therapy? 
c. What treated cells (or vector/DlA combination) will be given 
to patients in the attempt to administer gene therapy? Hew 
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? Hew 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? Hew long will patient follow-up 
continue? 
e. What are the major potential beneficial and adverse effects of 
treatment that you anticipate? What 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. 
f. If a treated patient dies, vhat special studies will be performed 
as part of the autepsy? 
(433] 
