HEPATOCELLULAR TRANSPLANTATION AND TARGETING GENETIC MARKERS TO HEPATIC CELLS 
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I these risks in detail [ ]. All transplant procedures, as well as the 
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I transfusion of blood and blood products, are associated with a risk of infection from 
j: the donor material. These risks will be the same as those associated with whole organ 
I transplantation and will be explained to you by the transplant team. The potential risk 
j associated with injection of liver cells include damage or clotting of the blood vessels 
which are necessary for blood flow to the liver. These complications could lead to 
j death . 
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j <par 7> The other risks of this protocol are those associated with the introduction 
I of the genetic marker into the transplanted cells. The genetic marker will be 
I introduced into liver cells by growing a portion of the liver cells in a laboratory and 
exposing them with a synthetic virus that permanently enters the cell and carries with 
it a gene that can be recognized in laboratory tests. This "marker" has no useful 
functions for your child, however, when liver cells containing this marker are infused 
into your child, researchers will be able to look for the marker in the transplanted 
j cells in liver biopsies and determine whether the liver cell injection is a success 
ij (whether the cells survive), where the infused cells reside, whether they are capable 
I of providing the normal functions of a liver cell, and how they withstand immune 
rejection. We believe that given the experimental nature of liver cell injection, this 
information is important for physicians and researchers to determine whether the liver 
cell injection is successful; whether it is a reasonable alternative to whole organ 
transplantation; and whether the same methods should be attempted again on other 
children. The risks involved in the use of the "marker gene" are those associated with 
gene transfer using synthetic (recombinant) viruses and are described in the following 
j paragraphs . 
<par 8> The virus which will be used in this study is a synthetic virus called a 
retrovirus. This virus has been designed to infect cells in culture, but it is 
defective in the functions which are required for spreading beyond a single cell or for 
causing human diseases. Like other "defective" viruses or. "live" viruses which are used 
routinely in clinical medicine as vaccines (for example the vaccines your child may 
have had for polio, measles, mumps, German measles, or influenza) this virus has been 
designed to carry out a medical function without causing disease in patients. The 
synthetic viruses which would be used in your child have been tested for safety, 
approved by the Food and Drug Administration for human research, and have been used 
previously in a small number of adult patients without any ill effects. You will be 
provided with a pamphlet written by the National Institutes of Health for patients which 
describes the methods for "gene therapy" which will be used in putting the genetic 
marker into the liver cells before transplant [ ] . 
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j <par 9> There are several theoretical risks associated with the use of these viruses: 
1) There is a possibility that the synthetic virus could naturally combine with 
other viruses in your child and give rise to a infectious virus. This has not 
been observed in laboratory studies or in adult patients. Also, viruses similar 
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