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Cystic
Fibrosis (CF)
Cystic Fibrosis (CF) mainly affects people's lungs
and digestive systems. A thick mucus
prevents normal breathing and digestion, leading to infections and
loss of the lungs' ability to function. Digestive enzyme
supplements and antibiotics may help, and periodically the airway
to the lungs may need clearing. Even so, half of the people with CF
die by the age of 30. CF affects one in 2000-3000 Caucasian babies.
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In 1989, the gene
causing Cystic Fibrosis (CF) was discovered. By 1993, gene therapy
trials to treat people with CF began. Today, researchers struggle
to develop a safe, effective gene therapy, as well as other,
more traditional therapies. Below is a timeline showing how
fast genetic research can sometimes lead to new attempts to
treat a disease. |
Letter
to Dr. Collins.
Courtesy of the
National Human Genome Research Institute |
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Below is a Timeline
Telling the Story of Developing a Gene Therapy for the Treatment of
Cystic Fibrosis: |
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| 1989 |
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| Celia
Hooper,
Journal of NIH Research, Nov-Dec.
1989. |
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Dr. Francis Collins' team at the
University of Michigan, and Drs. Lap-Chi Tsui and John Riordan's team
at the University of Toronto locate the CF gene. The protein
made from the instructions encoded by the gene is called CFTR (cystic
fibrosis transmembrane-conductance regulator). |
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| 1990 |
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Dr.
Michael Welsh's team at the University of Iowa demonstrated what the
protein called CFTR does in the human body. They added normal CFTR
to CF cells which had been cultured
in the laboratory. When normal CFTR was added, the chloride (salt)
transport between the cell
membranes began to function correctly. |
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| 1993 |
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| Dr.
Crystal's gene therapy trial. Courtesy of the National Heart,
Lung, and Blood Institute. |
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When the
safety and effectiveness of gene therapy had been proven as well as
it could be in the laboratory and during studies in animals, the first
human gene therapy trials for CF were conducted. Dr. Ronald Crystal,
then of the National Heart, Lung, and Blood Institute, used copies
of the normal CF gene to treat the lining of the nose and the lungs
of people with CF (in this picture Dr. Crystal is third from the right).
The trial tested how safe the procedure was for humans. Some people
experienced inflammation in their lungs.
Gene therapy trials to treat people with CF continue to take place
in several places. |
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