Monitoring After Discharge from the Hospital: After your liver cells have been infused back 
into your liver and you have left the hospital, we will need to monitor you closely for the effects 
of the corrected liver cells. For the 4 weeks following your discharge from the hospital, you 
will have to come back to the hospital for 2 days each week (each visit will last about one hour). 
After these first four weeks, you will have to come back to the hospital for visits every week for 
four months. After this time, the frequency of your visits will be determined by your medical 
condition with a minimum of one visit per year. Small amounts of blood (6 teaspoons or less) 
will be taken from you on each visit. We will also need to remove a small piece of your liver 
tissue in order to examine it for the corrected cells. This will be done about three months after 
the infusion of the corrected cells back into your liver. A doctor will place a needle through 
your skin into your liver and remove a tiny piece (the size of a pencil tip) of your liver. This 
would not require another operation. This procedure will be explained to you at the time it will 
be performed and your consent will be obtained separately. If you die for any reason, either 
during or after your participation in this study, we will request that an autopsy be performed. 
This will be necessary so that we could determine the cause of death and to examine the results 
of this therapy. 
Benefits: It is impossible to say that you will receive any benefit from this study. Similar 
studies in rabbits that also have this type of disease have shown that a similar form of therapy 
lowered the "fat" level in their blood for at least four months. This suggests that this therapy 
may lower your lipid levels, however, it is impossible to predict the likelihood that a human 
being will respond in the same manner as a rabbit. Following infusion of the corrected liver 
cells, you will be restarted on your previous lipid lowering therapy. Important information 
will be gained in this study that may benefit other patients with this disease or similar 
inherited diseases. 
Risks, Inconvenience and Discomforts: There are many possible risks and discomforts 
that may happen to you in this study. These will be explained in detail below. 
Initial Evaluation: Problems may occur from the blood drawing, which involves a needle stick, 
and may result in a bruise at the site of the stick. Very rarely a local infection may occur at the 
site of the needle stick that would require treatment with antibiotics or drugs to kill the 
bacteria causing the infection. Other inconveniences include the time required for this 
evaluation. 
Liver Surgery: Severe complications may result from the operation that removes part of your 
liver. It is possible that you would experience problems with the anesthesia that would affect 
and possibly cause damage to your heart and brain. It is very rare, but you may die from these 
problems with the anesthesia. Death from the surgery itself is a very rare complication (less 
than 0.1%) but this can occur. The surgery to remove part of your liver may also result in 
internal bleeding or an infection. This may require a second operation to correct the bleeding. 
There may also be damage to your liver that causes it to fail (this is also very rare; with less 
than a 1% chance occuring during the procedure). Pain from the surgery site may persist for 
long periods (several months) after the surgery, but this will diminish with time. Limitations 
to certain activities, such as exercise, will be necessary due to the surgical wound. These 
activities may not be started until you have complete healing of this area. Infection from the 
tube that was put into the vein that leads to your liver may also occur and this would require 
treatment with antibiotics or removal of the tube. 
.(initials) 
Recombinant DNA Research, Volume 15 
[205] 
