Attachment VI 
Section of Document Entitled 
"Points to Consider in the Design and Submission of 
Somatic Cell Gene Therapy Protocols" 
1 . Objectives and Rationale 
a. In a paragraph state concisely the overall objectives 
and rationale of the proposed study. 
b. Why was the particular disease selected? 
c. Is the natural history or range of expression of the 
disease known, so that assessment of the results of 
gene therapy will be unambiguous? 
d. What alternative therapies exist? In what groups of 
patients are the therapies effective, and how effective 
are they? 
Robert F. Murray 
LeRoy Walters 
November 12, 1984 
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