Contents 
Authors 2 
Contents 3 
Preface 4 
I. Description of Proposal 6 
A. Objectives and rationale of the proposed research 6 
B. Research design 14 
1. Structure and characteristics of the biological system 14 
a. Structure of the cloned DNA 14 
b. Structure of the material that will be administered 15 
(1) Viral particle preparation 15 
(2) Producer cells 18 
2. Precllnlcal studies 19 
a. Delivery system 19 
b. Expression 32 
c. Safety 45 
3. Clinical procedures 62 
4. Public health considerations 69 
5. Qualifications of Investigators 71 
C. Selection of patients 72 
D. Informed consent 75 
E. Privacy and confidentiality 83 
II. Special Issues 85 
III. Requested Documentation 86 
Appendix Contents 86 
References 90 
Recombinant DNA Research, Volume 12 
[3] 
