I 
HEPATOCELLULAR TRANSPLANTATION AND TARGETING GENETIC MARKERS TO HEPATIC CELLS 
to the synthetic virus we will use in this study are normally present in many 
species (including mice and cats), and no evidence of infection among humans has 
ever been found. As part of this protocol we will ask to obtain blood, urine, 
stool, and saliva samples to be sure that no infectious virus are formed. 
2) There is a possibility that when the synthetic virus enters the cell it may 
damage an essential function of that cell or even induce that cell to become 
cancerous. This has not been observed in laboratory studies or in adult 
patients. As part of this protocol we will ask to test your child for any of 
these complications using X-ray, ultrasound, and blood tests of liver function, 
obtaining blood tests which may indicate the presence of any malignancy, and 
biopsies of the blood cells in the bone marrow. 
3) There is a possibility that there may be long-term consequences of viral 
infection which have not yet been observed in laboratory or human studies. 
Accordingly, the investigators and the Texas Children's Hospital will make a 
commitment to participate in long-term follow-up for your child in conjunction 
with your personal physician. We will strongly advise you to participate in this 
long-term follow-up program either here at Texas Children's Hospital or through 
your personal physician. This follow-up will include routine health surveillance 
for possible complications of the synthetic virus as well as genetic counselling 
when your child is of child-bearing age. We would strongly advise that your 
child never donate blood or organs which might contain the synthetic virus . 
i; 
' 1 
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<par 10> You will be free to withdraw from this protocol at any time. If you do choose 
to withdraw from the study we will make every effort to arrange continuing medical care 
and surveillance required to ensure the safety of your child through the physician of 
your choice. 
<par 11> One of the potential benefits of this study is that the materials and methods | 
which will be used to introduce the "marker” gene into your child's liver cells are j 
identical to methods which may be used for "somatic gene therapy" in the future, ji 
Somatic gene therapy would be performed by infecting a patient's own liver cells with !' 
a synthetic virus which carried a gene capable of correcting the genetic defect in these | 
cells. ■, 'I 
<par 12> Many questions have been asked in recent years about the idea and safety of 
methods for somatic gene therapy, and many government and religious bodies in this 
country have considered how synthetic viruses should be used in medicine. Because of 
these questions, the use of synthetic viruses in human experiments is carefully 
regulated by the government. These committees believe it is important that you be 
informed that the introduction of "marker" genes into cells and transplantation of these 
cells into your child's liver is "novel" and "irreversible"; that long-term follow-up 
of you child by physicians and investigators at the Texas Children's Hospital is very 
important; that, because of the experimental nature of the transplant and gene marking 
procedure, if your child should die despite this therapy, you will be encouraged to 
consent to an autopsy; and that there may be considerable public interest in you and or 
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Recombinant DNA Research, Volume 14 
