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5.6) Theoretical Side Effects and Monitoring 
5.61) Theoretical Side Effects 
No side effects have been seen in animal 
studies nor the very limited human 
research. However, since this is a very 
new procedure, the risks may not be 
known. There are some theoretical risks 
to this procedure. First, even though 
the virus used to insert the gene into 
the bone marrow cells cannot grow and is 
considered harmless, it is possible that 
events could occur within a cell that 
cause the cell to become cancerous. It 
is also theoretically possible that the 
LNL-6 vector preparation used could be 
contaminated with replication-competent 
murine retrovirus. What, if any, affects 
replication-competent murine retroviruses 
have on humans is unknown (see Section 
7.4, Safety Issues). The inserted gene 
may produce a protein that inactivates 
certain antibiotics. These antibiotics 
are not commonly used to treat infections 
in humans. Many other antibiotics are 
available that will not be inactivated 
and these will be used in treating any 
bacterial infections that might occur. 
5.62) Monitoring for Adverse Reactions 
Side effects are not expected from this 
treatment. However, side effects from 
the bone marrow transplantation itself 
will be universal. When evaluating this 
treatment, we will need to separate side 
effects that would be considered normal 
for bone marrow transplantation from 
those attributable to the gene transfer. 
Side effects will be evaluated 
continually on each patient. After three 
patients have been entered, we will 
suspend accrual until each has received 
their 21 day work-up, in order to 
quantify side effects. If grade 3-4 
toxicity not attributable to the bone 
marrow transplant occurs, entry will be 
suspended and the FDA and OPRR of the NIH 
will be contacted immediately. The study 
will reactivate only if the PI and the 
two regulatory agencies agree that the 
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