Attachment II - Page 9 
9 
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 enployed 
for gene delivery and the rationale for their use. The following 
are specific points to be addressed: 
a. What is the structure of the cloned DtPi that will be used? 
(1) Describe the gene (genomic or cDNA) , the bacterial plasmid 
or phage vector, and the delivery vector (if any). Provide 
ccrrplete nucleotide sequence analysis or a detailed restriction 
enzyme map of the total construct. 
(2) What regulatory elements does the construct contain (e.g. , 
promoters , erhancers, polyadenylation sites, replication 
origins, etc.)? 
(3) Describe the steps used to derive the ENA construct. 
b. What is the structure of the material that will be administered 
to the patient? 
(1) Describe the preparation, structure, and ccnposition of the 
materials that will be given to the patient or used to 
treat the patient's cells. 
(a) If DNA, v^iat is the purity (both in terms of being a 
single DNA species and in terms of other contaminants)? 
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