B. Research design, anticipated risks and benefits 
1 . Structure and characteristics of the biological system 
Provide a full description of the methods and reagents to be employed 
for gene delivery and the rationale for their use. The following 
are specific points to be addressed: 
a. Vhat is the structure of the cloned CNA that will be used? 
(1) Describe the gene (genanic or cDNA) , the bacterial plasmid 
or phage vector, and the delivery vector (if any). Prcvide 
complete nucleotide sequence analysis or a detailed restriction 
enzyme map of the total construct. 
(2) Vhat regulatory elements does the construct contain (e.g., 
promoters, enhancers, polyadenylation sites, replication 
origins, etc.)? 
(3) Describe the steps used to derive the CNA construct. 
b. Vhat is the structure of the material that will be administered 
to the patient? 
(1) Describe the preparation, structure, and composition of the 
materials that will be given to the patient or used to 
treat the patient's cells. 
(a) If CNA, what is the purity (both in teams of being a 
single DNA species and in terns of other contaminants)? 
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