Human Gene Therapy Subcommittee - 7/30/90 
6. What degree of infection was obtained when cells were 
treated with a single cycle of virus, which is 
equivalent to the proposed NIH protocol? 
7. Figure 6. Why is there no diagnostic band in clone B56 
at 5.3 kilodaltons? 
8. Figure 2. Where are the controls? Where is the 
histogram showing staining with directly labeled 
ascites to demonstrate that the 4% staining is CD4- 
specific? What is the staining pattern with an 
antibody to CDS? How many CD4^ cells were detected 
when non- transfected cells were injected? How many 
CD4‘’’ cells were detected when the peripheral blood T 
lymphocytes, transfected with a control vector, were 
injected? 
9. Was human antibody to tetanus toxoid produced in 
addition to just immunoglobulin, since tetanus toxoid- 
specific T lymphocytes are present? 
Dr. Parkman summarized these questions as falling into three 
general areas: 
1. What is the origin of the immunologially functional 
cells? 
2. Was the fact that immune logically functional cells were 
present due to the fact that the ADA gene was inserted, 
or could an analogous degree of function be seen in the 
patient's peripheral blood? 
3. If the immunological function seen was a consequence of 
the insertion of the ADA gene, how diverse was the 
repertoire or spectrum of cells that were responding? 
Were they all derived from a limited number of cells or 
were they many different cells? 
Dr. Childress said he felt the conditions previously set for 
approval of the protocol were based on: whether there was some 
expectation that this therapy would work; whether it was safe; 
whether the use of children was acceptable; and finally issues of 
informed consent. 
Dr. Childress said he found the revised informed consent document 
well organized and said that the addition of headings had helped. 
He said it had been appropriately expanded but without providing 
excessive detail. It was generally concise and clear. However, 
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