d. What are the clinical endpoints of the study? Are there 
objective and quantitative measurements to assess the natural 
history of the disease? Will such measurements be used in 
following' your patients? How will patients be monitored to 
assess specific effects of the treatment on the disease? What 
is the sensitivity of the analyses? How frequently will follow-up 
studies be done? How long will patient follow-up continue? 
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? Canpare 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. 
f. If a treated patient dies, vhat special studies will be performed 
as part of the autopsy? 
4. Public-health considerations 
Describe any potential benefits and hazards of the proposed 
therapy to persons other than the patients being treated. 
Specifically: 
a. On what basis are potential public health benefits or hazards 
postulated? 
b. Is there a significant likelihood that the added DNA will 
spread from the patient to other persons or to the environment? 
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