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Inhaling Genes to Tackle Cystic Fibrosis

A thrilling new approach has been developed for targeted gene therapy that brought a cloud of excitement over the scientific community. You might have guessed it – the delivery of vaporized gene vectors to battle cystic fibrosis.

The prospect of gene therapy was first proposed before the first human coding sequence was even determined. However, a prescient report published in Science soon after questioned this novel approach in regards to patient safety. It outlined the construction of a safe viral gene delivery vector and efficient gene delivery as major concerns among many others. To this day, we are facing many of the same hurdles, but it is undeniable that gene transfection came a long way from where it started decades ago.

Results of numerous recent clinical trials show great promise and it seems that we are finally taking the helm of gene therapy as a clinical approach. It even convinced the editorial staff of Science to reconsider their initial stance, naming “the return of gene therapy” as one of the biggest scientific breakthroughs in 2009.

A shining example is a novel gene therapy treatment that takes a very different form, literally. It involves nebulising (fancy for vaporization) cationic liposoms and applying them to patients with cystic fibrosis through inhalation.

The CFTR protein is a channel protein that controls the flow of H2O and Cl- ions in and out of cells inside the lungs. When the CFTR protein is working correctly, as shown in Panel 1, ions freely flow in and out of the cells. However, when the CFTR protein is malfunctioning as in Panel 2, these ions cannot flow out of the cell due to a blocked channel. This causes Cystic Fibrosis, characterized by the buildup of thick mucus in the lungs.

The CFTR protein is a channel protein that controls the flow of H2O and Cl- ions in and out of cells inside the lungs. When the CFTR protein is working correctly, as shown in Panel 1, ions freely flow in and out of the cells. However, when the CFTR protein is malfunctioning as in Panel 2, these ions cannot flow out of the cell due to a blocked channel. This causes Cystic Fibrosis, characterized by the buildup of thick mucus in the lungs.

Cystic fibrosis is a genetic disorder that results in excessive build-up of mucus on epithelial surfaces of the lungs, caused by a lack in expression of a cell surface receptor called CFTR. This leads to severe infections, breathing problems and even sepsis to occur.

The novel approach aims to transfect the patien’s epithelial lung cells using vectors with functional CFTR genes packed inside the liposomes, enabling them to express the missing receptor. The study showed significant improvements in the experimental group while producing no sign of adverse effects. For a disease that currently holds no cure, this is a pretty big step in the right direction.

 

 

By Luka Zupančič, MSc, University of Applied Sciences Technikum Vienna

 

Original article: UK’s first cystic fibrosis gene therapy trial

Image source: Wikimedia Commons





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