Attachment VI - Page 4 
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qroup judges that it is appropriate to review somatic-cell gene therapy protocols 
at this time and to approve those protocols if they adequately address the 
questions raised in the following document. However, in recoc^ntion of the 
social concern that surrounds the general practice of himan gene therapy, the 
workinq group will, in its review of individual scmatic-cell proposals, also 
consider the following issues: (1) how strong is the evidence that somatic-cell 
gene therapy does not affect reproductive cells; (2) does somatic-cell gene 
therapy make it more likely that reconbinant ENA techniques will be employed 
for: (a) germ- line gene therapy; (b) the enhancenent of human capabilities 
throuqh genetic means; or (c) eugenic programs encouraged or even mandated by 
ooverrments? If so, what are the implications of proceeding with somatic-cell 
qene therapy? 
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 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. 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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