and related laboratory and animal experiments in order to define appropriate 
human applications of this energing 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 concisely the overall objectives and rationale of the proposed 
study. Please provide information on the following specific points: 
1. Vfriy 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. What objective and/or quantitative measures 
of disease activity are available? In your view, are the usual 
effects of the disease predictable enough to allow for meaningful 
assessment of the results of gene therapy? 
3. Is the protocol designed to prevent all nan ifes tat ions of the disease, 
to halt the progression of the disease after symptcms have begin 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? Wiat are their relative advantages and 
disadvantages as compared with the proposed gene therapy? 
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