Cystic Fibrosis Gene Therapy WisdomCard


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Research Notes on Cystic Fibrosis Gene Therapy


Cystic fibrosis gene therapy focuses on curing the disease by correcting the defective gene involved.1 The therapy shows promise as it treats the cause of the disease and not just the symptoms.1 The first experimental gene therapy treatment with a cystic fibrosis patient was conducted in 1993.1

Fast Facts:

  • The first treatment involved modifying a common cold virus as a method to deliver normal genes to the cystic fibrosis transmembrane conductance regulator (CFTR) cells in the airways of the lung.2
  • Researchers have studied nose drops and aerosol nebulizers as other delivery methods.2
  • Finding the best delivery system for the normal gene is the challenge researchers must solve.2


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Research Notes References

  1. 1.0 1.1 1.2 National Human Genome Research Institute: Gene Therapy Research Offers Promise of a Cure for Cystic Fibrosis
  2. 2.0 2.1 2.2 eMedTV: Cystic Fibrosis Gene Therapy

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