Attachment V - Page 2 
stated assumption, the working group judges that it is appropriate to 
review somatic-cell gene therapy protocols that may be presented at this 
time and to approve these protocols if they satisfactorily address the 
questions raised in the following document. However, in recognition of the 
social concern that surrounds the general practice of human gene therapy, 
the working group will, in its review of individual somatic-cell proposals, 
also consider the following issues: (1) is the proposed somatic-cell gene 
therapy a sufficient departure from existing medical therapies to require 
new risk-assessment and/or unusual precautions? (2) how strong is the 
evidence that the proposed somatic-cell gene therapy will not inadvertently 
affect reproductive cells? (3) does the proposed somatic-cell gene therapy 
contribute toward: (a) intentional germ-line gene therapy; (b) the enhancement 
of, rather than the correction of defects in, human capabilities through 
genetic means; or (c) governmental or other programs with eugenic objectives? 
Responses to the questions raised in these "Points to Consider" should be in 
the form of either written answers or references to specific sections of the 
protocol or other documentation which accompanies the proposal . 
I. Description of Proposed 
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. 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 
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