Cystic Fibrosis and Stem Cells

Cystic fibrosis (CF) is a genetic condition which affects the lungs and digestive system as they become clogged with thick sticky mucus.[1]

In order for a child to be born with cystic fibrosis, both parents must be a carrier of the faulty gene which causes it. When both parents carry the faulty gene, each child they have has 25% chance of having the condition, a 25% chance of being healthy, and a 50% chance of being a carrier.[2]

There is no cure for cystic fibrosis and current treatments are symptomatic. Treatments can include antibiotics for frequent lung infections, physiotherapy to help clear the airways of mucus, and drugs to expand the airways. In some cases a lung transplant may also be necessary.[3]

Cystic fibrosis is one of nine illnesses tested for on the newborn screening programme through the blood spot, heel prick, test.[5] Early diagnosis and treatment are critical to improve quality of life and life expectancy. A report in 2012 found that cystic fibrosis is progressively moving away from being to a childhood killer to an adult disease. Thanks to improvements in the care of patients, the average life expectancy for sufferers in the UK is now 43.5 years.[6]

Cystic Fibrosis Facts

  • It is estimated that 46.8 million people have dementia worldwide [4]
  • CF is most common in people of Northern European descent [3]
  • CF is the most common inherited disease in white people [4]
  • There are 9,000 people living with CF in the UK[3]
  • It is estimated that 1 in 2,500 babies are born with CF [3]
  • The average life expectancy in the UK for CF sufferers is 43.5 years of age [6]
  • There are currently 2 clinical trials investigating the application of stem cells for treatment [7]

Cystic Fibrosis and Stem Cells

Research at the University of Melbourne faculty of medicine aims to prove cord blood could be used to treat cystic fibrosis. The study suggests that umbilical cord blood could be used to restore lung function. While the research is in preliminary stages, the researchers hope to use sibling cord blood to treat the condition.[8]

The Cystic Fibrosis Trust has also funded a project to correct two rare mutations of the cystic fibrosis gene. The project will use induced pluripotent stem cells made from white blood cells along with gene repair and editing technology to repair the two rare “stop codon” mutations.

Once the researchers have fixed the mutation they will generate lung epithelial cells to confirm that normal function has been restored. It is hoped that these cells could then be used to replace the cells in the lungs of patients with cystic fibrosis in the future.[9]

References

  1. http://www.nhs.uk/conditions/Cystic-fibrosis/Pages/Introduction.aspx
  2. http://www.geneticalliance.org.uk/docs/translations/english/8-cf-t.pdf
  3. http://www.nhs.uk/conditions/Cystic-fibrosis/Pages/Introduction.aspx
  4. http://patient.info/doctor/cystic-fibrosis-pro
  5. http://www.nhs.uk/Conditions/pregnancy-and-baby/Pages/newborn-blood-spot-test.aspx
  6. http://cysticfibrosis.org.uk/news/registry-report-2012
  7. https://clinicaltrials.gov/ct2/results?term=cystic+fibrosis+stem+cells&Search=Search
  8. http://www.nzherald.co.nz/bay-of-plenty-times/news/article.cfm?c_id=1503343&objectid=11033787
  9. http://www.cysticfibrosis.org.uk/news/stem-cell-project

The information contained in this article is for information purposes only and is not intended to replace the advice of a medical expert. If you have any concerns about your health we urge you to discuss them with your doctor.

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