Findings recently published in the Stem Cell Research and Therapy journal show that diseased cells in the airways affected by cystic fibrosis could be replaced with healthy ones using cell transplantation therapy.
This therapy is a technique that uses stem cells. In this case, the researchers harvested adult stem cells from the airways of cystic fibrosis (CF) patients, corrected them with gene therapy, and then reintroduced them to the airways.
This method, according to Dr. Nigel Farrow, a Post-Doctoral Research Fellow from the University of Adelaide's Robinson Research Institute and one of the researchers involved in the study, would give space for the corrected cells to pass on their genetic material to their “daughter cells” -- the cells resulting from cell division – allowing the healthy cells to replicate in those airways.
In initial studies, the researchers tested this method on mouse airways by clearing out the existing surface cells and introducing adult stem cells with a marker gene as opposed to the corrective CF gene used in later studies.
Why does this matter?
Cystic fibrosis is a life-limiting, genetic disease that is caused by a defective CF gene. This gene causes the epithelial cells in patients' lungs and other parts of their bodies to produce a faulty version of the CFTR (cystic fibrosis transmembrane conductance regulator) protein, affecting the cell's ability to regulate the transportation of chloride ions across cell membranes.
This skews the balance of salt (sodium chloride) and water necessary for keeping the mucus coating in those parts of the body thin and able to carry out their functions and instead, causes the build-up of thick mucus that is hard to move.
In the lungs, this causes germs to remain trapped in the thick mucus and increases the likelihood of lung infections. People living with the disease must undergo regular physical therapy sessions to move the trapped mucus in their lungs.
Dr. Farrow says that if the technique in the study proves to be successful, it could help significantly improve the lives of people living with the chronic condition.
Other organs, such as the pancreas, can also be affected. In the pancreas of patients, thick mucus build-up stops nutrients from food being absorbed into the body, this can lead to poor growth and malnutrition.
CF in numbers
- Around 30,000 people in the United States have the are affected and 70,000 worldwide, according to the Cystic Fibrosis Foundation Patient Registry.
- The average lifespan for patients in the United States is 40. This is considered to be a vast improvement from the 1950s when children barely survived elementary school.
- The illness affects people who inherit two copies of the defective CF gene. This often happens when two carriers of one copy of the gene who don't have the condition have a child who inherits both copies. The likelihood that two carriers will have a child with the condition is 25 percent.
