Gene replacement in pigs ameliorates cystic fibrosis-associated intestinal obstruction

Cystic fibrosis (CF) is caused by mutations in CFTR and is characterized by dysfunction of the lungs, liver, pancreas, and intestines. Approximately 15% of babies with CF are born with an obstruction of the small intestine known as meconium ileus, frequently the first sign of CF. Unlike in humans, meconium ileus occurs in 100% of newborn CF pigs.

n this issue of the Journal of Clinical Investigation, Michael Welsh and colleagues at the University of Iowa demonstrate that transgenic expression of normal CFTR in the intestine of CF pigs alleviated meconium ileus. Over time, the pigs still exhibited other manifestations of CF, including liver and lung disease, reduced weight gain, and pancreatic destruction.

These findings provide insight into the pathophysiology of CF and indicate that tissue-specific, partial gene replacement can ameliorate intestinal symptoms of CF.

More information: Intestinal CFTR expression alleviates meconium ileus in cystic fibrosis pigs, J Clin Invest. doi:10.1172/JCI68867

Related Stories

Mucous breakthrough in mice holds promise for cystic fibrosis

date Jul 29, 2008

A London, Canada scientist studying cystic fibrosis (CF) has successfully corrected the defect which causes the overproduction of intestinal mucous in mice. This discovery by Dr. Richard Rozmahel, a scientist with the Lawson ...

Pigs provide clues on cystic fibrosis lung disease

date Apr 28, 2010

Aided by a new experimental model, scientists are a step closer to understanding how cystic fibrosis (CF) causes lung disease in people with the condition. The findings, published online April 28 in the journal Science Tr ...

Recommended for you

Niger battles deadly meningitis epidemic

date 9 hours ago

Parents cradling sick children in their arms streamed into a treatment centre in Niger's capital Niamey, the victims of a meningitis epidemic that has claimed over 100 lives and appears to be accelerating.

Long lasting anti-hemophilia factor safe in kids

date 13 hours ago

Children with hemophilia A require three to four infusions each week to prevent bleeding episodes, chronic pain and joint damage. The effect on quality of life can be significant, due to time and discomfort associated with ...

Missouri detective battles flesh-eating infection

date 18 hours ago

Friends and loved ones of Lee's Summit, Mo., Police Detective Joshua Ward are praying for the 34-year-old married father of three who, even after five surgeries in as many days, remained in critical condition Monday at St. ...

User comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.