MEDICAL 
RECORD 
CONTINUATION SHEET for either: 
NIH 2514-1, Consent to Participate in A Clinical Research Study 
NIH 2514-2, Minor Patient’s Assent to Participate in A Clinical 
Research Study 
STUDY NUMBER: 
CONTINUATION: page 2 of 6 page- 
stem cells prior to transplantation. The neoc ene will be inserted into some cells obtained from 
your bone marrow as well as some cells obtained from your blood stream (peripheral blood stem 
cells). 
It is important to understand that the introduction of this gene will not benefit you in any 
way. It is possible that this study may lead to improved bone marrow transplantation therapies 
for others in the future. The purpose of the study is to assess the safety of this procedure and to 
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determine if "retroviral-mediated gene transfer” will enable the successful transfer of the necA 
gene to patients' bone marrow and circulating blood cells. 
Evaluation and Procedures 
If you decide to participate in this study, special laboratory techniques will be used to insert 
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the neo^ gene into a portion of your bone marrow and circulating blood stem cells after they are 
harvested. These treated bone marrow and circulating cells are referred to as "marked” blood 
cells. A gene is part of a chromosome (hereditary material) that contains the information a cell 
needs to make proteins. By inserting a gene into your bone marrow and blood cells, we will be 
able to distinguish these marked cells from all other cells in your body. 
The following procedure will be used to insert the neo^ gene into your bone marrow and 
blood cells. Bone marrow cells will be obtained by a bone marrow harvest in the operating room 
using the regular procedure for harvest. This has been described to you in a separate consent 
document for autologous transplantation. We will not need to obtain any more bone marrow 
than is normally taken during bone marrow harvest. Therefore, there will be no additional 
needle aspirations or additional anesthesia time. Your circulating cells will be obtained by 
removing some blood through a vein, separating the immature blood cells of interest, and 
returning the remaining blood to you through a vein. This process is called apheresis and has 
also been explained to you. The major portion of the marrow and blood cells harvested will be 
handled in the usual fashion and will not be treated by retroviral-mediated gene transfer. A 
portion of the marrow and blood cells (no more than 30%) will undergo retroviral-mediated gene 
transfer. In this 30% portion, the cells to undergo the gene transfer will be separated from the 
other bone marrow and blood cells by attaching a special antibody to them and then physically 
removing only the cells that can pick-up the antibody. This procedure is called 
“immunoadsorption” and it allows us to separate the more immature stem cells out of your bone 
marrow and blood cells. These immature cells are the ones used for the gene transfer. The 
physical separation of the immature cells attached to the special antibody is done by a laboratory 
machine called an immunoadsorption column. The column to be used in this protocol is 
experimental and has been approved by the FDA for use in transplantation studies such as this. 
PATIENT IDENTIFICATION 
CONTINUATION SHEET for either: 
NIH-25 1 4-1 (10-84) 
NIH-251 4-2 (10-84) P.A.: 09-25-0099 
Recombinant DNA Research, Volume 16 
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