MEDICAL RECORD 
CONTINUATION SHEET for either: 
NIH 2514-1, Consent to' Participate In A Clinical Research Study 
NIH 2514-2, Minor Patient’s Assent to Participate In A Clinical Research Study 
STUDY NUMBER: CONTINUATION: page 3 of 8 pages 
other imaging studies, and physical examination to determine the 
status of your Gaucher disease and of your liver, kidneys and bone 
marrow. You will have 2-4 tablespoons of blood drawn. You will sign 
a separate consent form for the bone marrow aspiration and biopsy, 
which will be done with local anesthesia and has minimal risks, 
including pain, bleeding and infection at the biopsy site. 
Collection of Bone Marrow: The first part of the protocol involves 
removing a portion of your bone marrow. You will be brought to the 
operating room and be placed under general anesthesia. The doctors 
will then remove about one liter of bone marrow from the area on 
the back of your hip bone known as the posterior iliac crest. This 
is done with hollow needles that are placed through the skin, and 
then through the outer surface of the bone. The harvest takes one- 
two hours. Risks include bleeding or infection. You will need to 
stayin the hospital the night after the harvest, and you will 
experience pain over your hip bones. You will receive pain 
medication to relieve this pain if necessary. There is a small 
possibility that the bone marrow harvest will make you anemic 
enough to require a blood transfusion. You will not be entered 
onto this protocol if you are already severely anemic, and we will 
do everything possible to prevent the need for a transfusion of 
blood from another person, including returning the red blood cells 
collected during the bone marrow harvest to you. 
Stem Cell Purification: The bone marrow cells will be further 
purified after harvest in the laboratory by a procedure called 
"CD34 selection." The cells will be exposed to an antibody 
(protein) that sticks specifically to the surface of immature 
cells. The cells will then be run through a special column that 
separates the immature cells coated with antibody from the more 
mature cells. This antibody and column procedure is experimental, 
but has been safely used to purify stem cells from the bone marrow 
and blood of over 50 patients without any known side effects. 
Gene Transfer: The purified stem cells will be treated in the 
laboratory for 3-4 days in the presence of a "vector" designed to 
place the GC gene in the cells' genetic ^material (chromosomes) . 
Once the GC gene is in the cell, it is permanent, and the cell will 
pass the GC gene onto all daughter cells derived from that cell. 
The vector is a mouse virus which has been altered so it cannot 
reproduce itself. No side effects have ,been seen in over 50 
patients that have received cells treated with this type of vector, 
although no patients prior to this study have received cells 
treated with the GC vector. 
PATIENT IDENTIFICATION 
CONTINUATION SHEET for either: 
[832] 
NIH-2514-1 (10-84) 
N H f^e^o ( m5mant DNA Research, Volume j^f 0099 
