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Gene Therapy--The Future of Cystic Fibrosis Treatment?Search our Archive for the Latest News on Cystic Fibrosis Gene therapy for cystic fibrosis is not yet possible but impressive progress is being made in developing ways to treat the gene abnormality that causes cystic fibrosis. In the laboratory, scientists have been able to grow cells from the nasal passages of CF patients. By introducing the normal gene into these cells, researchers corrected the cells' chloride transport abnormality. The chloride defect has also been corrected in small regions in the nasal passages themselves by giving CF patients the normal gene in nose drops. Scientists are still looking for answers to many questions about gene
therapy. Some of these questions are: How should the gene be packaged? What are
the best ways to get the gene-containing package into the patient's lungs? What
will the long-term results of this treatment be? Can the abnormal chloride
transport be corrected in other parts of the body? How long will the correction
last? And, most importantly, can gene therapy cure or prevent the lung disease
in CF?
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