(2) Describe any other material to be used in preparation of 
the material bo be administered to the patient. For example, 
if a viral vector is proposed, what is the nature of the 
helper virus or cell line? If carrier particles are bo be 
used, what is the nature of these? 
2. Preclinical studies, including risk-assessment studies 
Describe the experimental basis (derived from tests in cultured 
cells and animals) for claims about the efficacy and safety of the 
proposed systerri for gene delivery. 
a. Laboratory studies of the delivery system 
(1) What cells are the intended recipients of gene therapy? 
If recipient cells are to be treated _in vitro and returned 
to the patient, how will the cells be characterized before 
and after treatment? Vhat is the theoretical and practical 
basis for assuming that only the treated cells will act as 
recipients? 
(2) Is the delivery systen efficient? Vhat percentage of the 
target cells contain the added DMA? 
(3) How is the structure of the added ENA sequences monitored 
and what is the sensitivity of the analysis? Is the added 
ENA extrachrcmosomal or integrated? Is the added ENA 
unrearranged? 
Recombinant DNA Research, Volume 1 1 
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