Attachment VII - Page 8 
and related laboratory and animal experiments in order to define appropriate 
huiran applications of this emerging technology. 
(12) Responses to the questions raised in these "Points to Consider" should be 
provided in the form of either written answers or references to specific 
sections of the protocol or its appendices. 
I. Description of Proposal 
A . Objectives and rationale of the proposed research 
State ccncisely the overall objectives and rationale of the proposed 
study. Please provide information on the follcv/ing specific points: 
1. Why is the disease selected for treatment by means of gene therapy 
a good candidate for such treatment? 
2. Describe the natural history and range of expression of the disease 
selected for treatment. In your view, are the usual effects of the 
disease predictable enough to allcv/ for meaningful assessment of 
the results of gene therapy? 
3. Is the protocol designed to prevent all manifestations of the disease, 
to halt the progression of the disease after symptoms have begun 
to appear, or to reverse manifestations of the disease in seriously 
ill victims? 
4. What alternative therapies exist? In what groups of patients are 
these therapies effective? What are their relative advantages and 
disadvantages as compared with the preposed gene therapy? 
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