costs, privacy, and long-term follow-up. 
1. How will the major points covered in Section I-A through I-C of 
this document be disclosed to potential participants in this study 
and/or parents or guardians in language that is understandable to 
them? 
The points outlined below would be explained in lay terms in the actual 
informed consent document which would be reviewed section by section with the 
patient/parents and child's advocate by one of the team physicians. 
2. How will the innovative character and the theoretical ly-possible 
adverse effects of gene therapy be discussed with patients and/or 
parents guardians? How will the potential adverse effects be 
compared with the consequences of the disease? What will be said 
to convey that some of these adverse effects, if they occur, could 
be irreversible? 
3. What explantation of the financial costs of gene therapy and any 
available alternative therapies will be provided to patients 
and/or parents or guardians? 
4. How will patients and/or their parents or guardians be informed 
that the innovative character of gene therapy may lead to great 
interest by the media in the research and in treated patients? 
5. How will patients and/or their parents or guardians be informed: 
a. That some of the procedures performed in the study may be 
irreversibl e? 
b. That following the performance of such procedures it would not 
be medically advisable for patients to withdraw from the 
study? 
c. That a willingness to cooperate in long-term follow-up (for at 
least three to five years) will be a precondition for 
participation in the study? 
d. That a willingness to permit an autopsy to be performed in the 
event of a patient's death following treatment is also a 
precondition for a patient's participation in the study? 
(This stipulation is included because an accurate 
determination of the precise cause of a patient's death would 
be of vital importance to all future gene therapy patients.) 
See suggested draft of Inormed Consent Document below 
Suggested Draft of an Informed Consent Document 
[76] 
Recombinant DNA Research, Volume 12 
