Gene Therapy for CF using Cationic Liposome Mediated Gene Transfer: Phase I Trial 
every three (3) months. 
PROCEDURES WHICH WELL BE PERFORMED 
AS PART OF THE RESEARCH STUDY 
If I agree to participate in this study, the following procedures will be performed: 
1. A complete physical examination, including chest x-rav. sinus x-rays and 
pulmonary function tests with arterial blood gases will be performed prior to the 
administration of the normal CF gene. Pulmonary function tests and blood gases 
will also be performed once following administration of the normal CF gene. X- 
rays will also be repeated after the gene is given. 
2. Blood samples (approximately 2 tsp each) will be taken 10-12 times over the five- 
month period of the study. The type of defect in my CF gene will be evaluated. 
These tests will also evaluate the function of vital organs (for example liver and 
kidneys) and blood counts. Sputum cultures, oximetries, urinalyses, and 
electrocardiogram will also be performed as part of this study. A pregnancy test 
(females) or sperm count (males) may also be performed. 
3. Administration of the normal CF gene . This will be accomplished in a hospital 
bed in The Children’s Hospital Clinical Research Center. On the morning of gene 
administration, I will be asked to lie on my side and a small flexible tube (about 
the thickness of a kite string) will be placed 2-4" inside one nostril. About 1/2 tsp 
of a solution containing lipid and a normal CF gene will be very slowly dripped 
into my nose over a period of about 30 minutes. I will then lie on my side for 
two hours (during which time I can watch TV) after the gene is given in order to 
give the gene time to enter the cells inside my nose. Late in the afternoon of the 
same day, I will be asked to lie on my opposite side, and a similar solution which 
contains no CF gene will be administered in my other nostril. The reason for 
using both nostrils (one which receives the normal CF gene and one which does 
not) will be to help prove that any desirable effects which are observed are truly 
due to the normal CF gene, since they should occur only in the nostril which 
receives the normal gene. 
4. Nasal potential difference measurements. The nasal potential difference 
measurement is performed in order to evaluate the movement of salts (such as 
sodium and chloride) across the tissue which lines the inside of the nose. Because 
individuals with CF have an abnormal nasal potential difference measurement, my 
potential difference measurement will be performed several times over the weeks 
before and after I am treated with the normal CF gene. The potential difference 
will be measured both in my nostril which receives the normal CF gene, and in 
the nostril which does not receive the gene, in order to verify that any changes 
which are observed are due to the presence of the normal CF gene in the cells 
lining the nose. 
The potential difference measurement is made by placing a needle under 
the skin of the forearm and then inserting a small piece of flexible tubing (again 
about the diameter of a kite string) between 2-6" into the nostril. The tubing will 
Recombinant DNA Research, Volume 18 
[847] 
