The document would be derived from the following outline which is 
based on Document per Orientation Manual of the Institute Clinical Review 
Subpanel. Nov.. 1983 - Consent to participate in a clinical research study, 
adult patient or parent for minor patient. Since the initial patients might 
very well be infants, they would be treated at Memorial Sloan Kettering Cancer 
Center's Pediatric Bone Marrow Transplantation Unit (no child under two years 
of age Is treated by a procedure involving general anesthesia at NIH). 
Therefore, the document would be written to conform with the Memorial Sloan 
Kettering Informed Consent requirements. 
INTRODUCTION 
We Invite your child to take part In a research study. It is Important 
that you read and understand several general principles: 
a) Taking part in the study is entirely voluntary. 
b) Personal benefit may not result from participation, but knowledge 
may be gained that would benefit others. 
c) You may withdraw from the study at any time without penalty or loss of 
any benefits to which you are otherwise entitled but it would not be 
medically advisable to withdraw at all, and certainly not before a minimum of 3 
to 5 years. 
The nature of the study, the risks, inconveniences, discomforts, and 
other pertinent information about the study are discussed below. You are urged 
to discuss any questions you have with the staff members who explain them to 
you. 
THE NATURE OF THE STUDY: 
This study attempts for the first time to treat children suffering from a 
genetic disease by inserting into some of their cells a normal copy of the 
defective gene which is responsible for their illness. 
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