Attachment II - Page 13 
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(e) Has a protoool similar to the one proposed for a clin- 
ical trial been carried out in non -human prirrates and/ 
or other animals? What were the results? Specifically, 
is there any evidence that the retroviral vector has 
recombined with any endogenous or other viral sequences 
in the anirrals? 
(2) If a non- retroviral delivery system is used: What animal 
studies have been done to determine if there are pathological 
or other undesirable consequences of the protocol (including 
insertion of DNA into cells other than those treated, 
particularly germ line cells)? Hew long have the animals 
been studied after treatment? What 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 frem 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? 
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