Explain how gene therapy might be used to threat cystic fibrosis?

Question

Its for my biology class.

Answer 1

Cystic fibrosis is the result of mutations in the cystic fibrosis transconductance regulator (CTFR) protein, which is a member of the ABC family of membrane transporters.  It behaves like an ion channel.

In cystic fibrosis, the CFTR is mutated such that it is either non-functional, or is degraded before reaching the cell membrane, where it belongs.

You only need one good copy of the gene for CFTR to avoid getting cystic fibrosis (i.e., it's a recessive disorder).  So if you can use gene therapy to insert even one good copy of the gene in a significant number of respiratory epithelial cells, the symptoms of cystic fibrosis should cease.

Answer 2

Gene therapy offers great promise for life-saving treatment for CF patients since it targets the cause of CF rather than just treating symptoms. Gene therapy for CF had its start in 1990, when scientists successfully corrected faulty CFTR genes by adding normal copies of the gene to laboratory cell cultures.

In 1993, the first experimental gene therapy treatment was given to a patient with CF. Researchers modified a common cold virus to act as a delivery vehicle - or "vector"- carrying the 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.

But finding the best delivery system for transporting normal CFTR genes is only one problem that scientists must solve to develop an effective treatment for CF. Scientists must also determine the life span of 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 cystic fibrosis 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. But scientists hope that the technologies being developed for lung cells will be adapted to treat other organs affected by CF.