Infusion of the liver cells : There are several risks from the infusion of cells. It is possible that 
infusion of cell would cause an infection, a blood clot, or possible bleeding. These complications 
may result in liver failure, and may require your child to undergo a liver transplant. The clots 
in the blood vessels near the liver may make liver transplantation difficult to perform. There 
are also risks involved with giving your child cells that have been genetically corrected. 
Although the mouse virus that is used to insert the gene for the normal LDL receptor into your 
child's liver cells has been inactivated, it is possible that events may occur within your child's 
liver cells that may result in the production of a new infectious virus or cancer of the liver. 
Because the technique of placing normal genes into cells is very new, it is also possible that this 
therapy may cause unforeseen problems. 
Period after leaving the hospital: The risks during this time involve the possibility of your 
child's body recognizing the new LDL receptor as foreign. His/her body may try to reject these 
cells. This may result in the destruction of the infused, corrected liver cells. The function of 
the rest of your child's liver should not be affected. There may also be the previously mentioned 
risks of blood drawing. 
Confidentiality of Research Records: The principal investigator will keep a confidential 
record of your child's participation, which will be maintained and secured in the Clinical 
Research Center of the University of Michigan. Representatives of the National Institutes of 
Health, The Food and Drug Administration, or other regulatory agencies outside the University 
of Michigan will have access to your child's records. Your child's privacy will be protected by 
withholding his/her identity during any recording and reporting of this research. 
Alternatives to Participation: Your child's participation in this study is entirely 
voluntary and vou mav withdraw vour child from the study at anv time. A decision not to 
participate will in no way jeopardize your child's health care, and he/she will continue to 
receive other therapies for his/her familial hypercholesterolemia as prescribed by your 
child's doctor. Alternative therapies for elevated blood "fats" involve taking medicine to lower 
the "fats" or to repeatedly siphon "fats" from your child's blood (plasmapheresis). Liver 
transplantation has also been shown to correct the defect in liver function in familial 
hypercholesterolemia. The physicians involved in this study can further explain these 
alternative forms of treatment and their risks and benefits to your child. 
Cost to You and Your Child: There will be no cost to you or your child for his/her 
participation in this study. Any costs resulting from care that your child requires as part of 
this study or because of complications from this study will be provided through the Clinical 
Research Center of the University of Michigan. 
Compensation for Illness or Injury: You and your child will receive no compensation for 
any physical injury or illness that results from his/her participation in this study, but as 
previously stated, your child will receive free treatment for these complications. 
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