Cystic Fibrosis Home > Cystic Fibrosis Gene Therapy

With gene therapy, treatment targets the cause of cystic fibrosis rather than just treating the symptoms. Although the first gene therapy experiments have involved lung cells, scientists hope that these technologies will be adapted to treat other organs affected by cystic fibrosis.

An Overview of Gene Therapy for Cystic Fibrosis

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

Gene therapy for cystic fibrosis 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 This Type of 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.
Written by/reviewed by:
Last reviewed by: Arthur Schoenstadt, MD
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