Gene Therapy: Cystic Fibrosis and how to treat it

Cystic Fibrosis is an inherited disease caused by irregular genes and you are born with it. It also means that people carrying the gene (but not necessarily with Cystic Fibrosis) can pass it to their children.

Cystic Fibrosis is caused by a defect in the CTFR gene. This gene controls the production of a protein that controls cell transport of chlorine ions. This disrupts the natural water potential and the mucus becomes thick, so the cilia can't move it. This results in blockages.

Cystic Fibrosis doesn't only affect the lungs, it causes problems for the entire body.

• The bile duct may become blocked, and leading to cirrhosis.
• Enzymes from the pancreas are not secreted which causes incomplete digestion.
• Infertility in both men and women.

The basic principle of gene therapy is to give patients copies of the correct gene, so they can make the protein by synthesis.

There are two vectors that are able to transport the CTFR gene.

1. 1
   A harmless virus, shown on the left, will deposit the gene into cells as viruses naturally do

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2. 2
   A liposome (see here) which can be inhaled, and will hopefully pass the gene through the cells.

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The problems with either of the methods of gene therapy are twofold. Firstly, it isn't permanent since epithelial cells for example have a short life and are constantly being renewed, so the treatment would have to be performed regularly - and this would be expensive. Also, since Cystic Fibrosis is a disease that affects the entire body, it isn't possible to treat everything, and while the lungs may be temporarily cured, the pancreas will not function properly and the sufferer will still need to take enzyme supplements.

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Gene Therapy: Cystic Fibrosis and how to treat it. (2017). In ScienceAid. Retrieved Jun 4, 2023, from https://scienceaid.net/biology/genetics/therapy.html

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