An Overview of Cystic Fibrosis Gene Therapy
Cystic fibrosis gene therapy offers great promise for life-saving treatment because it targets the cause of
cystic fibrosis rather than just treating the symptoms of cystic fibrosis.
History of Cystic Fibrosis Gene Therapy
Cystic fibrosis gene therapy began in 1990, when scientists successfully corrected faulty cystic fibrosis transmembrane conductance regulator (CFTR) genes. They did this by adding normal copies of the gene to laboratory cell cultures.
In 1993, the first experimental CF gene therapy treatment was given to a patient with cystic fibrosis. Researchers modified a
common cold virus to act as a delivery vehicle by carrying normal genes to the CFTR cells in the airways of the lung.
Subsequent studies have tested other methods of gene delivery, such as: fat capsules, synthetic vectors, nose drops, or drizzling cells down a flexible tube to CFTR cells lining the airways of lungs. Researchers are now testing aerosol delivery using nebulizers.
Challenges of Cystic Fibrosis Gene Therapy
Finding the best delivery system for transporting normal CFTR genes is only one challenge that scientists must solve to develop an effective
treatment for cystic fibrosis. Scientists must also:
- Determine the life span of the affected lung cells
- Identify the "parent cells" that produce CFTR cells
- Find out how long treatment should last and how often it needs to be repeated.
The first CF gene therapy experiments have involved lung cells because these cells are readily accessible and because lung damage is the most common, life-threatening problem in CF patients. However, scientists hope that the technologies being developed for lung cells will be adapted to treat other organs affected by cystic fibrosis.
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