5. While your cells are being given the new ribozyme gene in the laboratory and allowed 
to multiply (a few days to weeks), you will be kept informed regularly about the 
schedule of what needs to be done next. You will be notified when the cells are ready to 
be put back into you. You will be admitted to the hospital or the UCSD Treatment 
Center and a needle (an intravenous line or TV.") will be inserted in your vein. Your 
genetically modified cells will then be infused back into you through the needle over a 
period of a few hours. 
6. Follow-up studies. Because this is a new and experimental treatment, we will be 
observing you closely during and after the treatment to determine if there are side 
effects to the therapy. We will also closely monitor the effects of this treatment on 
your immune system. We will do this is through a combination of regularly talking to 
you about any symptoms or problems you may have, examining you in the clinic and 
by doing a variety of blood tests. It will take about a year of such visits to complete 
your participation in the study, at first every week or so for a month and then about 
every month or so. Small amounts of blood for testing will be drawn at each visit. 
Possible Risks, Discomforts and Side Effects 
Participation in this study may involve some added risks or discomforts. 
These include: 
1 . You may experience discomfort or bruising from blood drawing and intravenous 
infusions. Sometimes people get lightheaded or even faint after blood drawing. There 
is also the remote possibility of infection at the blood drawing site or I.V. site. 
2. Although the DNA that will be inserted in your cells is considered safe, there is 
a remote chance that inserting the gene into the DNA of your cells could cause cancer 
to develop, which would create additional problems and may require additional 
treatments. Laboratory studies and previous studies of similar gene therapy in people 
suggest that this possibility is very low. However, this is a new procedure and we do 
not know whether cells could become abnormal after a long time. 
3. The inserted DNA has a gene that can cause a few antibiotics to be inactivated in the 
test tube. This gene will not likely be active in any appreciable amount in your body 
and many other antibiotics that are not inactivated by it will be available for treating 
any potential bacterial infections. Therefore it is unlikely to significantly interfere 
with giving you effective antibiotics for any infections you may develop in the future. 
4. Although many efforts will be made to ensure no bacteria, viruses or other kinds of 
infections get into your cells while they are being genetically modified outside your 
body, a small possibility exists that such contamination could occur and cause 
harmful or even fatal infection when the cells are put back. 
5. Although previous animal and human studies suggest it is unlikely, there is a 
possibility that having a new gene in some cells of your body could provoke a response 
by your immune system against those and other cells in your body. 
If you are injured as a result of participation in this research, the University of 
California will provide any medical care you need to treat those injuries-except when 
they are a consequence of research procedures designed to benefit you directly. The 
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