Assent for the Performance of Gene Transfer Therapy for Familial 
Hypercholesterolemia 
You have been asked to take part in this study because you have an illness called familial 
hypercholesterolemia. This illness causes your liver to work poorly and not remove the fats 
from your blood. This can lead to hardening of the arteries, heart disease and poor circulation. 
We hope to fix the liver cells in your body by first removing part of your liver, placing the 
normal gene for the lipid clearing protein into these liver cells and injecting these cells back 
into your body. 
This is a very serious study and involves many risks to you. You will have an operation to 
remove part of your liver and will have to be in the hospital for at least three weeks. After you 
are discharged from the hospital, you will have to come back to the hospital office very often for 
at least five months after the surgery. You will suffer pain and discomfort from the surgery for 
at least several weekSj and there is even a small risk that this study will hurt your liver or 
even kill you. It is not even clear that you can expect any benefit from this study. You can, 
however, decide to quit and stop this study at any time and it will not affect how the doctors take 
care of you. 
It is important that you let us know that you understand all of the things we have told you and 
ask any questions you have. If you have any second thoughts about taking part in this study, 
please tell us and we will let you stop the study. 
Your signature on this paper tells us that we have told you all the problems with this study, and 
you have had all your questions answered and are willing to take part in the study. You can 
always quit and stop the study if you change your mind. 
Participant 
Date 
Witness 
Date 
Recombinant DNA Research, Volume 15 
[213] 
