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Massachusetts General Hospital 
MEDICAL RESEARCH CONSENT FORM, Page 1 
Title of Project: Adenovirus -Mediated Gene Transfer for Cystic 
Fibrosis: Safety of Single Lcbar Administration in the Lung 
Principal Investigator: Allen Lapev. 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 infections 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 the 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. 
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Recombinant DNA Research, Volume 20 
