The patient would have all organ systems monitored for possible side 
effects of the therapy and would be followed for the remainder of their lives 
with the testing frequency being decreased as warranted by the patient's 
clinical status. Blood samples for testing could be taken by the patient's 
family physician and shipped to NIH for analysis. 
Besides testing for the ADA gene and following the patient's immune 
status, the patient may require monitoring for clinical factors (nutrition, 
infections, anemia, etc.) which are related to the patient's underlying disease 
but are not related to gene therapy. These parameters would be monitored as 
clinically indicated and appropriate intervention would be undertaken as 
warranted. 
e. What are the major potential beneficial and adverse effects of 
treatment that you anticipate? What measures will be taken in 
an attempt to control or reverse these adverse effects if they 
occur? Compare the probability and magnitude of potential 
adverse effects on patients with the probability and magnitude 
of deleterious consequences from the disease if gene therapy Is 
not performed. 
(1) Beneficial Effects . Several major and minor beneficial effects of the 
procedure are anticipated. ADA(-)SCID if left untreated is lethal. The major 
expectation for gene therapy is to cure the patient's underlying disease. If 
the patient responds to the therapy as hoped, he/she would no longer be at 
increased risk of mortality from everyday infectious exposures and should be 
able to lead a perfectly normal life. The other members of the family would 
also benefit from the restoration of a normal family situation. 
Even a partial restoration of immunity would benefit the patient's 
well-being in many ways. Most children with SCID are malnourished because of 
chronic low-grade intestinal infections that do not permit the patient to 
adequately absorb oral intake. Restoration of some immune function would help 
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Recombinant DNA Research, Volume 12 
