would require you to lie quietly in the scanner tube for approximately 20 to 30 minutes 
while you are being studied. 
The amount of blood drawn will be limited to approximately 30 cc (six teaspoons) per 
drawing. There will be a total of 10 blood drawings in the first year. 
RISKS/DISCOMFORTS: 
The risk posed to you from the gene transfer virus itself is not precisely known. That is the 
main question the study is attempting to answer. The natural AAV virus does infect 
humans but appears to cause no adverse effects. The genetically engineered gene transfer 
virus has been studied extensively in animals and has been found to be safe in that setting. 
There is a potential risk of seeing complications in humans which did not occur in animals, 
however. 
It is possible that the virus itself, or the bronchoscopy procedure, could trigger acute 
wheezing or coughing which might impair your breathing. We will be prepared for this 
possibility with oxygen, breathing treatments (bronchodilator nebulizations), and other 
supportive equipment. It is also possible that you could gradually develop an immune or 
inflammatory reaction in your lungs which could resemble asthma (wheezing) or 
pneumonia. Since you probably already have some lung disease due to CF, this could 
impair your long-term health and well-being. There is also a possibility of long-term side 
effects from the virus inserting its DNA into the cells of your lungs. Although this does 
not occur normally with AAV in people or animals, it is theoretically possible that the gene 
transfer virus could damage the DNA in your lung cells and put you at risk for lung cancer. 
There is also a small potential risk of your shedding the gene transfer virus. We will be 
culturing your secretions periodically to determine if this is occurring, and if it does occur, 
we will give you specific instructions on how to prevent spread of the virus to others in 
your home environment. 
The other major risks relate to the sedation for the bronchoscopy, and the bronchoscopy 
procedure itself. Intravenous sedation, particularly in patients with a history of lung 
disease, carries with it the risk, that due to an adverse reaction to the medications, you 
could experience a drop in your oxygen levels or stop breathing for a long enough period 
of time that a tube might need to be passed through your mouth into your windpipe to allow 
for support of your breathing efforts. It would be more likely that you might need some 
extra oxygen by mask or nasal cannula (tube) to help you through any difficulties due to 
sedation. If oxygen or a breathing tube were required due to the sedation, this would be 
expected to be a temporary situation and would probably reverse itself when the sedation 
began to wear off. The bronchoscopy, in addition to triggering wheezing or coughing as 
mentioned above, could contribute to temporary drops in your oxygen concentration. 
Other complications that could occur would be nosebleeds, fevers, or bleeding from the 
lung itself. Nosebleeds could also be seen following nasal brushing or nasal TEPD 
measurements. As a safety precaution, the above studies will be performed in the presence 
of both the study doctor and an anesthesiologist, both of whom will be monitoring closely 
for potential problems and be ready to intervene and stop the procedure if necessary. 
BENEFITS: 
THE BENEFITS TO YOUR OWN HEALTH FROM PARTICIPATING IN THIS 
INITIAL STUDY ARE LIKELY TO BE MINIMAL TO NONE. Many of the patients 
involved in the study will receive a very low dose of gene transfer virus, and as such will 
not be likely to show much effect. The study will target the virus to only one lobe of the 
lung, so that even if that lobe were very effectively treated it would not likely have a great 
Recombinant DNA Research, Volume 20 
