lining of the airway (called the bronchial tube) in the same way we measured 
the voltage in your nose. We will place about one-half of a teaspoon of salt 
solution in the right lung. These procedures take about 60-90 minutes. We will 
be with you as you wake up. You may need extra oxygen as you wake up. In order 
to keep your lungs as healthy as possible during your stay in the hospital, 
you will receive antibiotics by a needle in a vein. You will also continue to 
receive chest physical therapy, just as you would if you had been admitted for 
pneumonia . 
When you are awake after the lung and blood studies you will be allowed to 
return to your room. We will test the samples we took from your blood, nose 
mucus, lung fluid, and stool to find out if you have one of several human 
"cold" viruses. It takes about 3 or 4 days before we have the results of 
these tests. If the tests show that you have a cold virus, we will discharge 
you from the hospital and you would not receive the gene transfer. We would 
ask you to return to the hospital one month later and have to do the tests for 
cold viruses again to see if we can give you the CFTR gene using the AAV 
virus . 
If the tests show that you do not have cold viruses, we will take more blood 
for new tests. We will repeat the nose and lung examinations while you are 
asleep This time, though, we would not put a salt solution in your nose and 
lung. Instead, we will place a small amount of the altered virus that contains 
the working gene into your nose and lung. The virus that we will use is called 
AAV. It is a human virus that normally enters the lung, but is not known to 
cause any disease. The AAV virus we will put into you has been changed 
(altered) to make sure it cannot multiply itself. We have added the working CF 
gene to this altered AAV virus. 
We have studied the altered AAV virus in the laboratory and in animals. Our 
studies in the laboratory and in animals make us think that it is safe to give 
to people. We think the altered AAV virus with the working CFTR gene will help 
CF patients make the protein needed to clear mucous from their lungs. This 
appears to happen in animals, but we do not know if this will work the same 
way in people. 
After the altered virus is added to your nose and lung, you will stay in the 
hospital for two weeks. During this time, you will be required to stay in 
respiratory isolation so that no one else can possibly be exposed to the 
altered virus. You will need to stay in your room and visitors will be 
required to wear masks, gloves, and gowns when they visit you. You will be 
able to continue to use your usual medicines and treatments while you are in 
the hospital. You will not be able to take any experimental drugs while you 
are in the hospital. 
We plan to perform a minimum of 4 and a maximum of 9 bronchoscopy examinations 
on you as part of this study. There will be one bronchoscopy 4 days before you 
receive the altered virus. We will give you the altered virus with the 
bronchoscope. After you receive the altered virus, there will be up to 7 
bronchoscopies over the following year, at 10, 30, 60, 90, 180, 270, and 365 
days. If we cannot find signs of the altered virus in your lungs on two 
successive bronchoscopies, we will not do any further bronchoscopies or nasal 
voltage tests. We will continue the other planned tests. We will also do other 
studies 1, 3, 10, and 14 days after we give you the altered virus. On these 
study days, you will have more blood drawn for tests, have lung function 
tests, and we will examine your nose and lungs. These are the same tests you 
had before you received altered virus. Not all of the tests will be done on 
each study day. 
[142] 
Recombinant DNA Research, Volume 20 
