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NEW ENGLAND MEDICAL CENTER 
INFORMED CONSENT FORM 
Version 3.5 
PROJECT TITLE: 
Adenovi rus -mediated gene transfer for 
Cystic Fibrosis: Safety of administration 
to the lung. 
II. Aerosol Administration 
PRINCIPAL INVESTIGATOR: Henry L. Dorkin, M.D. 
CO- INVESTIGATOR : 
Joseph Oren, M.D. 
PURPOSE 
During the course of treatment for your disease, you have learned 
that cystic fibrosis is a serious genetic disease that causes the 
airways of the lung to fill, with thick mucus. This situation 
leads to frequent infection's which in turn cause destructive 
changes in the lung and increasing problems with breathing. 
We now know that the underlying defect in CF is a mutation in a 
gene that is responsible for producing a protein called the 
cystic fibrosis transmembrane conductance regulator, or CFTR. 
This protein regulates salt and fluid balance in the airways of 
the lungs; when the protein is not normal, excessive thick mucus 
is produced. Although antibiotics and chest physical therapy 
help control infection and clear secretions, no treatment is yet 
available to correct the underlying defect. Thus, despite all 
efforts, the disease in the lung usually worsens over time. 
The purpose of this study is not to provide a cure or permanent 
treatment for participants at this time, but to study new 
approaches which may eventually lead to better treatment for lung 
disease in CF patients. This study will examine the safety and 
possibility of correcting t:ie basic genetic defect by replacing 
the defective CFTR gene in the cells of your airway with a normal 
CFTR gene . 
STUDY DESIGN AND SAFETY FACTORS 
In this study, the normal gene that produces the CFTR protein 
will be transferred to the cells of your airway by a virus which 
has been altered so that it cannot reproduce normally. The 
virus, known as adenovirus 2, is a common virus that is found in 
human airways and in its natural state most often produces a 
cold. The changes in this virus limit, but do not eliminate, its 
ability to reproduce, spread, and cause disease. The modified 
virus containing the normal CFTR gene is called Ad2/CFTR-2, and 
is prepared at Genzyme Corporation in Framingham, Massachusetts. 
Ad2/CFTR-2 has been shown to be able to enter the cells that form 
the lining of the airway and deposit the gene so that normal CFTR 
is produced by the cell. Ad2/CFTR-2 is an investigational agent 
Recombinant DNA Research, Volume 20 
