Detailed Description of Participation: Your participation in this study involves a 
number of very complicated steps and procedures. These will be explained individually in the 
order that they will be done. You should be aware that you can withdraw at any point in the 
study without jeopardizing your future care at the hospital. 
Initial Evaluation : You will undergo an extensive evaluation to determine if you are eligible to 
participate in this study. We will conduct a medical history and physical exam, blood tests 
(which will remove about 4 to 6 teaspoons of your blood with a needle), urine analysis, X-rays 
and electrocardiogram. Because patients with familial hypercholesterolemia sometimes 
experience hardening of the arteries of the heart, we may perform a cardiac catheterization. If 
you require this, that procedure and its risks will be explained thoroughly and your consent 
will be obtained separately. 
Liver Surgery: You will be admitted to the hospital one week before the operation that removes 
part of your liver. During this one week time period, the levels of "fats" in your blood will be 
monitored daily. Six teaspoons-of blood will be drawn from you every day during this week. 
We will next perform the surgery that removes part of your liver You will receive 
general anesthesia (drugs) that puts you to sleep. A tube will be inserted through your mouth 
into your lungs and a machine will breathe for you during the surgery. The surgeon will make a 
4 inch cut across your abdomen just below your ribs. The surgeon will then remove the left 
part (15-20%) of your liver, being careful not to damage the rest of the liver. A tube will 
then be placed in a blood vessel which leads to your liver. This tube will be used three days 
later to infuse the corrected liver cells (see below). The surgeon will then close your wound by 
suturing (sewing) or stapling it shut. We will awaken you and watch you closely in the 
recovery room to determine if any complications occur (explained below). After you are stable, 
we will take you back to your hospital room so that you can recover. You will be able to leave 
the hospital two weeks after this surgery, although you may experience some pain and 
discomfort for a much longer period. 
Reinfusion of the Corrected Liver Cells : Three days after your surgery, you will be taken to a 
special room where doctors will use fluoroscopy (X-rays) to make sure that the tube is still in 
the blood vessel that leads to your the liver. The doctors will then take the liver cells (that have 
been given the normal gene for the LDL receptor) and infuse them through this tube. They will 
then remove the tube. You will remain in the hospital for 7-10 days after infusion of the 
corrected liver cells so that we can monitor you for possible complications of the therapy 
(explained below). The total length of your stay in the hospital for the liver surgery and 
infusion of the liver cells will be approximately 3 weeks. It is possible that there may be 
technical problems with either isolation of the cells or placement of the normal gene into your 
liver cells. If this occurs, we may be unable to reinfuse your liver cells back into your liver 
and the study would have to be terminated at this point. 
.(initials) 
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Recombinant DNA Research, Volume 15 
